[go: up one dir, main page]
More Web Proxy on the site http://driver.im/

MX359327B - Composiciones y sus usos para tratar y prevenir la enfermedad de huntington. - Google Patents

Composiciones y sus usos para tratar y prevenir la enfermedad de huntington.

Info

Publication number
MX359327B
MX359327B MX2014010455A MX2014010455A MX359327B MX 359327 B MX359327 B MX 359327B MX 2014010455 A MX2014010455 A MX 2014010455A MX 2014010455 A MX2014010455 A MX 2014010455A MX 359327 B MX359327 B MX 359327B
Authority
MX
Mexico
Prior art keywords
disease
compositions
methods
treating huntington
treating
Prior art date
Application number
MX2014010455A
Other languages
English (en)
Other versions
MX2014010455A (es
Inventor
C Miller Jeffrey
J Rebar Edward
Steve Zhang H
Original Assignee
Sangamo Biosciences Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Sangamo Biosciences Inc filed Critical Sangamo Biosciences Inc
Publication of MX2014010455A publication Critical patent/MX2014010455A/es
Publication of MX359327B publication Critical patent/MX359327B/es

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • C07K14/4703—Inhibitors; Suppressors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/70—Fusion polypeptide containing domain for protein-protein interaction
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/70—Fusion polypeptide containing domain for protein-protein interaction
    • C07K2319/71—Fusion polypeptide containing domain for protein-protein interaction containing domain for transcriptional activaation, e.g. VP16
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Toxicology (AREA)
  • Immunology (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Physics & Mathematics (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Psychology (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

La presente invención se refiere a métodos y composiciones para tratar o prevenir la enfermedad de Huntington.
MX2014010455A 2012-02-29 2013-02-28 Composiciones y sus usos para tratar y prevenir la enfermedad de huntington. MX359327B (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201261605028P 2012-02-29 2012-02-29
PCT/US2013/028348 WO2013130824A1 (en) 2012-02-29 2013-02-28 Methods and compositions for treating huntington's disease

Publications (2)

Publication Number Publication Date
MX2014010455A MX2014010455A (es) 2015-02-10
MX359327B true MX359327B (es) 2018-09-25

Family

ID=49083290

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2014010455A MX359327B (es) 2012-02-29 2013-02-28 Composiciones y sus usos para tratar y prevenir la enfermedad de huntington.
MX2018009444A MX374399B (es) 2012-02-29 2013-02-28 Composiciones y sus usos para tratar y prevenir la enfermedad de huntington.

Family Applications After (1)

Application Number Title Priority Date Filing Date
MX2018009444A MX374399B (es) 2012-02-29 2013-02-28 Composiciones y sus usos para tratar y prevenir la enfermedad de huntington.

Country Status (14)

Country Link
US (7) US8841260B2 (es)
EP (1) EP2820159B1 (es)
JP (3) JP6490426B2 (es)
KR (1) KR102084539B1 (es)
CN (2) CN108285491B (es)
AU (1) AU2013225950B2 (es)
BR (1) BR112014021104B1 (es)
CA (1) CA2865011C (es)
HK (1) HK1203566A1 (es)
MX (2) MX359327B (es)
NZ (1) NZ629427A (es)
RU (1) RU2639277C2 (es)
WO (1) WO2013130824A1 (es)
ZA (1) ZA201406209B (es)

Families Citing this family (169)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2734621B1 (en) 2011-07-22 2019-09-04 President and Fellows of Harvard College Evaluation and improvement of nuclease cleavage specificity
ES2991004T3 (es) 2011-12-22 2024-12-02 Harvard College Métodos para la detección de analitos
WO2014163886A1 (en) 2013-03-12 2014-10-09 President And Fellows Of Harvard College Method of generating a three-dimensional nucleic acid containing matrix
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
JP6490426B2 (ja) 2012-02-29 2019-03-27 サンガモ セラピューティクス， インコーポレイテッド ハンチントン病を治療するための方法および組成物
UA118014C2 (uk) 2012-05-25 2018-11-12 Те Ріджентс Оф Те Юніверсіті Оф Каліфорнія Спосіб модифікації днк-мішені
AU2013355214B2 (en) 2012-12-06 2017-06-15 Sigma-Aldrich Co. Llc Crispr-based genome modification and regulation
IL239317B (en) 2012-12-12 2022-07-01 Broad Inst Inc Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
EP3434776A1 (en) 2012-12-12 2019-01-30 The Broad Institute, Inc. Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
EP2825654B1 (en) 2012-12-12 2017-04-26 The Broad Institute, Inc. Crispr-cas component systems, methods and compositions for sequence manipulation
WO2014099744A1 (en) 2012-12-17 2014-06-26 President And Fellows Of Harvard College Rna-guided human genome engineering
CA2904960A1 (en) 2013-03-14 2014-10-02 University Of Florida Research Foundation, Inc. Di-amino acid repeat-containing proteins associated with als
US20140315985A1 (en) 2013-03-14 2014-10-23 Caribou Biosciences, Inc. Compositions and methods of nucleic acid-targeting nucleic acids
EP4428141A3 (en) 2013-03-15 2024-12-18 The General Hospital Corporation Rna-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci
US10760064B2 (en) 2013-03-15 2020-09-01 The General Hospital Corporation RNA-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci
US11332719B2 (en) * 2013-03-15 2022-05-17 The Broad Institute, Inc. Recombinant virus and preparations thereof
WO2014204578A1 (en) 2013-06-21 2014-12-24 The General Hospital Corporation Using rna-guided foki nucleases (rfns) to increase specificity for rna-guided genome editing
JP2016522679A (ja) 2013-04-04 2016-08-04 プレジデント アンド フェローズ オブ ハーバード カレッジ ＣＲＩＳＰＲ／Ｃａｓ系を用いたゲノム編集の治療的使用
US20140356956A1 (en) 2013-06-04 2014-12-04 President And Fellows Of Harvard College RNA-Guided Transcriptional Regulation
JP6621738B2 (ja) 2013-06-04 2019-12-18 プレジデント アンド フェローズ オブ ハーバード カレッジ Ｒｎａ誘導性転写制御
JP6738728B2 (ja) 2013-06-17 2020-08-19 ザ・ブロード・インスティテュート・インコーポレイテッド 肝臓ターゲティングおよび治療のためのＣＲＩＳＰＲ−Ｃａｓ系、ベクターおよび組成物の送達および使用
SG11201510286QA (en) 2013-06-17 2016-01-28 Broad Inst Inc Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components
ES2777217T3 (es) 2013-06-17 2020-08-04 Broad Inst Inc Suministro, modificación y optimización de sistemas de guía en tándem, métodos y composiciones para la manipulación de secuencias
CN105492611A (zh) 2013-06-17 2016-04-13 布罗德研究所有限公司 用于序列操纵的优化的crispr-cas双切口酶系统、方法以及组合物
EP3725885A1 (en) 2013-06-17 2020-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
SG10201710488TA (en) 2013-06-17 2018-01-30 Broad Inst Inc Delivery, Engineering and Optimization of Systems, Methods and Compositions for Targeting and Modeling Diseases and Disorders of Post Mitotic Cells
WO2015006498A2 (en) 2013-07-09 2015-01-15 President And Fellows Of Harvard College Therapeutic uses of genome editing with crispr/cas systems
BR112016000571B1 (pt) 2013-07-10 2023-12-26 President And Fellows Of Harvard College Métodos in vitro para modular a expressão e para alterar um ou mais ácidos nucleicos alvo em uma célula simultaneamente com a regulação da expressão de um ou mais ácidos nucleicos alvo em uma célula, bem como célula de levedura ou bactéria compreendendo ácidos nucleicos
US10563225B2 (en) 2013-07-26 2020-02-18 President And Fellows Of Harvard College Genome engineering
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
JP7083595B2 (ja) * 2013-10-25 2022-06-13 セレクティス 高反復モチーフを備えたｄｎａ配列を効率的かつ特異的に標的とするレアカットエンドヌクレアーゼの設計
WO2015065964A1 (en) 2013-10-28 2015-05-07 The Broad Institute Inc. Functional genomics using crispr-cas systems, compositions, methods, screens and applications thereof
KR102380245B1 (ko) 2013-11-07 2022-03-30 에디타스 메디신, 인코포레이티드 지배적인 gRNA를 이용하는 CRISPR-관련 방법 및 조성물
US10369201B2 (en) * 2013-11-11 2019-08-06 Sangamo Therapeutics, Inc. Methods and compositions for treating Huntington's disease
US10787684B2 (en) 2013-11-19 2020-09-29 President And Fellows Of Harvard College Large gene excision and insertion
US9074199B1 (en) 2013-11-19 2015-07-07 President And Fellows Of Harvard College Mutant Cas9 proteins
EP2878667A1 (en) * 2013-11-29 2015-06-03 Institut Pasteur TAL effector means useful for partial or full deletion of DNA tandem repeats
KR20160089526A (ko) * 2013-12-12 2016-07-27 더 브로드 인스티튜트, 인코퍼레이티드 입자 전달 성분을 이용한 표적 장애 및 질병에 대한 ｃｒｉｓｐｒ-ｃａｓ 시스템 및 조성물의 전달, 이용 및 치료 적용
SG10201804976YA (en) 2013-12-12 2018-07-30 Broad Inst Inc Delivery, Use and Therapeutic Applications of the Crispr-Cas Systems and Compositions for Genome Editing
CA2932439A1 (en) 2013-12-12 2015-06-18 The Broad Institute, Inc. Crispr-cas systems and methods for altering expression of gene products, structural information and inducible modular cas enzymes
US20150166985A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting von willebrand factor point mutations
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
MX374530B (es) 2013-12-12 2025-03-06 Broad Inst Inc Suministro, uso y aplicaciones terapeuticas de los sistemas y composiciones crispr-cas para actuar sobre hbv y trastornos y enfermedades virales.
WO2015089486A2 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems
EP3080258A1 (en) * 2013-12-12 2016-10-19 The Broad Institute, Inc. Compositions and methods of use of crispr-cas systems in nucleotide repeat disorders
WO2015122967A1 (en) 2014-02-13 2015-08-20 Clontech Laboratories, Inc. Methods of depleting a target molecule from an initial collection of nucleic acids, and compositions and kits for practicing the same
EP3114227B1 (en) 2014-03-05 2021-07-21 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa
US11141493B2 (en) 2014-03-10 2021-10-12 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
CA2948728A1 (en) 2014-03-10 2015-09-17 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10)
US11339437B2 (en) 2014-03-10 2022-05-24 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
EP3929279A1 (en) * 2014-03-18 2021-12-29 Sangamo Therapeutics, Inc. Methods and compositions for regulation of zinc finger protein expression
EP3122880B1 (en) 2014-03-26 2021-05-05 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating sickle cell disease
JP2017515819A (ja) * 2014-05-08 2017-06-15 サンガモ セラピューティクス， インコーポレイテッド ハンチントン病を処置するための方法および組成物
CA2947622A1 (en) 2014-05-13 2015-11-19 Sangamo Biosciences, Inc. Genome editing methods and compositions for prevention or treatment of a disease
US9970001B2 (en) 2014-06-05 2018-05-15 Sangamo Therapeutics, Inc. Methods and compositions for nuclease design
WO2016007839A1 (en) 2014-07-11 2016-01-14 President And Fellows Of Harvard College Methods for high-throughput labelling and detection of biological features in situ using microscopy
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
US9616090B2 (en) 2014-07-30 2017-04-11 Sangamo Biosciences, Inc. Gene correction of SCID-related genes in hematopoietic stem and progenitor cells
EP2982758A1 (en) 2014-08-04 2016-02-10 Centre Hospitalier Universitaire Vaudois (CHUV) Genome editing for the treatment of huntington's disease
WO2016033246A1 (en) 2014-08-27 2016-03-03 Caribou Biosciences, Inc. Methods for increasing cas9-mediated engineering efficiency
US11021718B2 (en) 2014-10-01 2021-06-01 The General Hospital Corporation Methods for increasing efficiency of nuclease-induced homology-directed repair
GB201418965D0 (es) 2014-10-24 2014-12-10 Ospedale San Raffaele And Fond Telethon
WO2016073955A2 (en) 2014-11-06 2016-05-12 President And Fellows Of Harvard College Cells lacking b2m surface expression and methods for allogeneic administration of such cells
US10570395B2 (en) 2014-11-14 2020-02-25 Voyager Therapeutics, Inc. Modulatory polynucleotides
WO2016094867A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Protected guide rnas (pgrnas)
EP3230452B1 (en) 2014-12-12 2025-06-11 The Broad Institute, Inc. Dead guides for crispr transcription factors
WO2016094874A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Escorted and functionalized guides for crispr-cas systems
EP3702456A1 (en) 2014-12-24 2020-09-02 The Broad Institute, Inc. Crispr having or associated with destabilization domains
TN2017000354A1 (en) 2015-02-10 2019-01-16 Genzyme Corp VARIANT RNAi
CA2982966C (en) 2015-04-24 2024-02-20 Editas Medicine, Inc. Evaluation of cas9 molecule/guide rna molecule complexes
US10179918B2 (en) 2015-05-07 2019-01-15 Sangamo Therapeutics, Inc. Methods and compositions for increasing transgene activity
US10968426B2 (en) 2015-05-08 2021-04-06 President And Fellows Of Harvard College Universal donor stem cells and related methods
US10509045B2 (en) 2015-05-29 2019-12-17 University Of Florida Research Foundation, Incorporated Methods for diagnosing Huntington's disease
WO2016205759A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Engineering and optimization of systems, methods, enzymes and guide scaffolds of cas9 orthologs and variants for sequence manipulation
EP3436575A1 (en) 2015-06-18 2019-02-06 The Broad Institute Inc. Novel crispr enzymes and systems
EP3929287A3 (en) 2015-06-18 2022-04-13 The Broad Institute, Inc. Crispr enzyme mutations reducing off-target effects
US9580727B1 (en) 2015-08-07 2017-02-28 Caribou Biosciences, Inc. Compositions and methods of engineered CRISPR-Cas9 systems using split-nexus Cas9-associated polynucleotides
US9926546B2 (en) 2015-08-28 2018-03-27 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
US9512446B1 (en) 2015-08-28 2016-12-06 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
MX391052B (es) * 2015-09-23 2025-03-21 Sangamo Therapeutics Inc Represores de htt y usos de estos.
DK3350327T3 (en) 2015-10-23 2019-01-21 Caribou Biosciences Inc CONSTRUCTED CRISPR CLASS-2-NUCLEIC ACID TARGETING-NUCLEIC ACID
EP3365357B1 (en) 2015-10-23 2024-02-14 President and Fellows of Harvard College Evolved cas9 proteins for gene editing
HK1252878A1 (zh) 2015-11-03 2019-06-06 President And Fellows Of Harvard College 用於含有基質的三維核酸的體積成像的方法和設備
WO2017143207A1 (en) 2016-02-18 2017-08-24 The Penn State Research Foundation GENERATING GABAergic NEURONS IN BRAINS
US11512311B2 (en) 2016-03-25 2022-11-29 Editas Medicine, Inc. Systems and methods for treating alpha 1-antitrypsin (A1AT) deficiency
US10940161B2 (en) 2016-04-04 2021-03-09 University Of Florida Research Foundation, Incorporated Manipulation of EIF3 to modulate repeat associated non-ATG (RAN) translation
CN116200465A (zh) 2016-04-25 2023-06-02 哈佛学院董事及会员团体 用于原位分子检测的杂交链反应方法
IL262784B2 (en) 2016-05-18 2023-10-01 Voyager Therapeutics Inc Modulatory polynucleotides
KR102427379B1 (ko) 2016-05-18 2022-08-02 보이저 테라퓨틱스, 인크. 헌팅톤 질환을 치료하기 위한 조성물 및 방법
US11124805B2 (en) 2016-07-13 2021-09-21 Vertex Pharmaceuticals Incorporated Methods, compositions and kits for increasing genome editing efficiency
US11566263B2 (en) 2016-08-02 2023-01-31 Editas Medicine, Inc. Compositions and methods for treating CEP290 associated disease
CA3032699A1 (en) 2016-08-03 2018-02-08 President And Fellows Of Harvard College Adenosine nucleobase editors and uses thereof
US11661590B2 (en) 2016-08-09 2023-05-30 President And Fellows Of Harvard College Programmable CAS9-recombinase fusion proteins and uses thereof
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
JP7256739B2 (ja) * 2016-09-07 2023-04-12 サンガモ セラピューティクス， インコーポレイテッド 肝臓遺伝子のモジュレーション
CA3039928A1 (en) 2016-10-14 2018-04-19 President And Fellows Of Harvard College Aav delivery of nucleobase editors
AU2017367722B2 (en) 2016-12-01 2024-02-01 Sangamo Therapeutics, Inc. Tau modulators and methods and compositions for delivery thereof
US9816093B1 (en) 2016-12-06 2017-11-14 Caribou Biosciences, Inc. Engineered nucleic acid-targeting nucleic acids
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
US11268093B2 (en) 2017-01-03 2022-03-08 Rula Zain-Luqman Therapeutic method for huntington's disease
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
US11542496B2 (en) 2017-03-10 2023-01-03 President And Fellows Of Harvard College Cytosine to guanine base editor
EP3596217A1 (en) 2017-03-14 2020-01-22 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
CA3057192A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
US11345911B2 (en) 2017-04-17 2022-05-31 University Of Florida Research Foundation, Incorporated Regulation of RAN translation by PKR and eIF2A-P pathways
JP2020517301A (ja) 2017-04-20 2020-06-18 イージェネシス，インコーポレイテッド 遺伝子改変動物の作成方法
CN119491003A (zh) 2017-05-05 2025-02-21 沃雅戈治疗公司 治疗亨廷顿病的组合物和方法
EP3622070A2 (en) 2017-05-10 2020-03-18 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
WO2018209320A1 (en) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
RS63202B1 (sr) 2017-06-30 2022-06-30 Inscripta Inc Automatizovani postupci, moduli, instrumenti i sistemi za obradu ćelija
WO2019023680A1 (en) 2017-07-28 2019-01-31 President And Fellows Of Harvard College METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE)
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
WO2019060726A1 (en) * 2017-09-22 2019-03-28 Genzyme Corporation Variant rnai
AU2018342105B2 (en) 2017-09-26 2023-11-16 University Of Florida Research Foundation, Incorporated Use of metformin and analogs thereof to reduce ran protein levels in the treatment of neurological disorders
CN111372650A (zh) 2017-09-30 2020-07-03 因思科瑞普特公司 流通式电穿孔仪器
KR20200121782A (ko) 2017-10-16 2020-10-26 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
WO2019143677A1 (en) 2018-01-17 2019-07-25 Vertex Pharmaceuticals Incorporated Quinoxalinone compounds, compositions, methods, and kits for increasing genome editing efficiency
JP7466448B2 (ja) 2018-01-17 2024-04-12 バーテックス ファーマシューティカルズ インコーポレイテッド Ｄｎａ－ｐｋ阻害剤
MA51619A (fr) 2018-01-17 2021-04-14 Vertex Pharma Inhibiteurs de la protéine kinase dépendante de l'adn
US20190233816A1 (en) 2018-01-26 2019-08-01 Massachusetts Institute Of Technology Structure-guided chemical modification of guide rna and its applications
KR20200132924A (ko) 2018-03-14 2020-11-25 에디타스 메디신, 인코포레이티드 혈색소병증 치료를 위한 시스템 및 방법
EP3781587A4 (en) 2018-04-18 2022-03-30 Sangamo Therapeutics, Inc. ZINC FINGER PROTEIN COMPOSITIONS FOR THE MODULATION OF HUNTINGTINE (HTT)
EP3797160A1 (en) 2018-05-23 2021-03-31 The Broad Institute Inc. Base editors and uses thereof
CN116144653A (zh) * 2018-06-21 2023-05-23 陕西杆粒生物科技有限公司 一种抗凋亡的杆状病毒表达载体
WO2020006126A1 (en) * 2018-06-27 2020-01-02 Altius Institute For Biomedical Sciences Nucleic acid binding domains and methods of use thereof
CA3115158A1 (en) * 2018-10-02 2020-04-09 Sangamo Therapeutics, Inc. Engineered genetic modulators
KR20210070344A (ko) 2018-10-02 2021-06-14 상가모 테라퓨틱스, 인코포레이티드 타우 단백질의 조정을 위한 방법 및 조성물
EA202190688A1 (ru) 2018-10-09 2021-06-07 Дзе Юниверсити Оф Бритиш Коламбиа Композиции и системы, содержащие трансфекционно-компетентные везикулы, не содержащие органических растворителей и детергентов, и связанные с ними способы
WO2020076976A1 (en) 2018-10-10 2020-04-16 Readcoor, Inc. Three-dimensional spatial molecular indexing
US12281338B2 (en) 2018-10-29 2025-04-22 The Broad Institute, Inc. Nucleobase editors comprising GeoCas9 and uses thereof
US12264323B2 (en) 2018-12-17 2025-04-01 The Broad Institute, Inc. CRISPR CPF1 direct repeat variants
CA3124110A1 (en) 2018-12-17 2020-06-25 The Broad Institute, Inc. Crispr-associated transposase systems and methods of use thereof
JP7542912B2 (ja) * 2019-01-15 2024-09-02 サンガモ セラピューティクス， インコーポレイテッド Ｈｔｔリプレッサーおよびその使用
BR112021018607A2 (pt) 2019-03-19 2021-11-23 Massachusetts Inst Technology Métodos e composições para editar sequências de nucleotídeos
KR20220003561A (ko) 2019-04-23 2022-01-10 상가모 테라퓨틱스, 인코포레이티드 염색체 9 오픈 리딩 프레임 72 유전자 발현의 조정제 및 그의 용도
JP2022539758A (ja) * 2019-06-28 2022-09-13 ザ・ペン・ステイト・リサーチ・ファウンデイション ハンチントン病を治療するための方法および材料
US20220267732A1 (en) 2019-08-01 2022-08-25 Sana Biotechnology, Inc. Dux4 expressing cells and uses thereof
CA3148370A1 (en) 2019-08-23 2021-03-04 Sana Biotechnology, Inc. Cd24 expressing cells and uses thereof
EP4051787A1 (en) 2019-11-01 2022-09-07 Sangamo Therapeutics, Inc. Gin recombinase variants
CN114728018B (zh) * 2019-11-01 2024-07-19 阿尔尼拉姆医药品有限公司 亨廷顿(HTT)iRNA药剂组合物及其使用方法
TW202132566A (zh) 2019-11-01 2021-09-01 美商聖加莫治療股份有限公司 用於治療或預防溶體貯積病之鋅指核酸酶變異體
KR20220158046A (ko) 2020-03-25 2022-11-29 사나 바이오테크놀로지, 인크. 신경계 장애 및 병태의 치료를 위한 면역저하성 신경 세포
IL297761A (en) 2020-05-08 2022-12-01 Broad Inst Inc Methods and compositions for simultaneously editing two helices of a designated double-helix nucleotide sequence
US20230190871A1 (en) 2020-05-20 2023-06-22 Sana Biotechnology, Inc. Methods and compositions for treatment of viral infections
CA3188664A1 (en) 2020-08-13 2022-02-17 Sonja SCHREPFER Methods of treating sensitized patients with hypoimmunogenic cells, and associated methods and compositions
US20220049303A1 (en) 2020-08-17 2022-02-17 Readcoor, Llc Methods and systems for spatial mapping of genetic variants
RU2748997C1 (ru) * 2020-12-11 2021-06-02 Федеральное государственное автономное образовательное учреждение высшего образования "Российский национальный исследовательский медицинский университет имени Н.И. Пирогова" Министерства здравоохранения Российской Федерации (ФГАОУ ВО РНИМУ им. Н.И. Пирогова Минздрава России) Универсальный эпигеномный репрессор транскрипции целевых генов в клетках человека
EP4271404A2 (en) 2020-12-31 2023-11-08 Sana Biotechnology, Inc. Methods and compositions for modulating car-t activity
AU2022283291A1 (en) 2021-05-27 2023-11-02 Sana Biotechnology, Inc. Hypoimmunogenic cells comprising engineered hla-e or hla-g
CA3225283A1 (en) 2021-07-14 2023-01-19 Sonja SCHREPFER Altered expression of y chromosome-linked antigens in hypoimmunogenic cells
WO2023019227A1 (en) 2021-08-11 2023-02-16 Sana Biotechnology, Inc. Genetically modified cells for allogeneic cell therapy to reduce complement-mediated inflammatory reactions
WO2023019203A1 (en) 2021-08-11 2023-02-16 Sana Biotechnology, Inc. Inducible systems for altering gene expression in hypoimmunogenic cells
US20250127820A1 (en) 2021-08-11 2025-04-24 Sana Biotechnology, Inc. Genetically modified cells for allogeneic cell therapy to reduce instant blood mediated inflammatory reactions
WO2023019226A1 (en) 2021-08-11 2023-02-16 Sana Biotechnology, Inc. Genetically modified cells for allogeneic cell therapy
IL310691A (en) 2021-08-11 2024-04-01 Sana Biotechnology Inc Genetically modified primary cells for allogeneic cell therapy
WO2023069790A1 (en) 2021-10-22 2023-04-27 Sana Biotechnology, Inc. Methods of engineering allogeneic t cells with a transgene in a tcr locus and associated compositions and methods
AU2022422147A1 (en) 2021-12-23 2024-07-04 Sana Biotechnology, Inc. Chimeric antigen receptor (car) t cells for treating autoimmune disease and associated methods
WO2023154578A1 (en) 2022-02-14 2023-08-17 Sana Biotechnology, Inc. Methods of treating patients exhibiting a prior failed therapy with hypoimmunogenic cells
JP2025508720A (ja) 2022-02-17 2025-04-10 サナ バイオテクノロジー，インコーポレイテッド 遺伝子操作されたｃｄ４７タンパク質及びその使用関連出願の相互参照
WO2023173123A1 (en) 2022-03-11 2023-09-14 Sana Biotechnology, Inc. Genetically modified cells and compositions and uses thereof
WO2024151541A1 (en) 2023-01-09 2024-07-18 Sana Biotechnology, Inc. Type-1 diabetes autoimmune mouse
WO2024229302A1 (en) 2023-05-03 2024-11-07 Sana Biotechnology, Inc. Methods of dosing and administration of engineered islet cells
WO2024243236A2 (en) 2023-05-22 2024-11-28 Sana Biotechnology, Inc. Methods of delivery of islet cells and related methods
WO2025004001A1 (en) 2023-06-30 2025-01-02 Takeda Pharmaceutical Company Limited Htt repressors and uses thereof
WO2025106739A1 (en) * 2023-11-14 2025-05-22 Nchroma Bio, Inc. Compositions and methods for epigenetic regulation of htt expression

Family Cites Families (102)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US789538A (en) 1904-11-11 1905-05-09 Colin E Ham Dumb-bell.
US4217344A (en) 1976-06-23 1980-08-12 L'oreal Compositions containing aqueous dispersions of lipid spheres
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
US4186183A (en) 1978-03-29 1980-01-29 The United States Of America As Represented By The Secretary Of The Army Liposome carriers in chemotherapy of leishmaniasis
US4261975A (en) 1979-09-19 1981-04-14 Merck & Co., Inc. Viral liposome particle
US4485054A (en) 1982-10-04 1984-11-27 Lipoderm Pharmaceuticals Limited Method of encapsulating biologically active materials in multilamellar lipid vesicles (MLV)
US4501728A (en) 1983-01-06 1985-02-26 Technology Unlimited, Inc. Masking of liposomes from RES recognition
US4946787A (en) 1985-01-07 1990-08-07 Syntex (U.S.A.) Inc. N-(ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US5049386A (en) 1985-01-07 1991-09-17 Syntex (U.S.A.) Inc. N-ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)Alk-1-YL-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4897355A (en) 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US4774085A (en) 1985-07-09 1988-09-27 501 Board of Regents, Univ. of Texas Pharmaceutical administration systems containing a mixture of immunomodulators
US5422251A (en) 1986-11-26 1995-06-06 Princeton University Triple-stranded nucleic acids
US4837028A (en) 1986-12-24 1989-06-06 Liposome Technology, Inc. Liposomes with enhanced circulation time
US5176996A (en) 1988-12-20 1993-01-05 Baylor College Of Medicine Method for making synthetic oligonucleotides which bind specifically to target sites on duplex DNA molecules, by forming a colinear triplex, the synthetic oligonucleotides and methods of use
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
AU7979491A (en) 1990-05-03 1991-11-27 Vical, Inc. Intracellular delivery of biologically active substances by means of self-assembling lipid complexes
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5420032A (en) 1991-12-23 1995-05-30 Universitge Laval Homing endonuclease which originates from chlamydomonas eugametos and recognizes and cleaves a 15, 17 or 19 degenerate double stranded nucleotide sequence
US5356802A (en) 1992-04-03 1994-10-18 The Johns Hopkins University Functional domains in flavobacterium okeanokoites (FokI) restriction endonuclease
US5487994A (en) 1992-04-03 1996-01-30 The Johns Hopkins University Insertion and deletion mutants of FokI restriction endonuclease
US5436150A (en) 1992-04-03 1995-07-25 The Johns Hopkins University Functional domains in flavobacterium okeanokoities (foki) restriction endonuclease
US5792632A (en) 1992-05-05 1998-08-11 Institut Pasteur Nucleotide sequence encoding the enzyme I-SceI and the uses thereof
US5587308A (en) 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
US6242568B1 (en) 1994-01-18 2001-06-05 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
WO1995019431A1 (en) 1994-01-18 1995-07-20 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
US6140466A (en) 1994-01-18 2000-10-31 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
CA2186118C (en) 1994-03-23 2010-10-19 Richard W. Hanson Compacted nucleic acids and their delivery to cells
US5585245A (en) 1994-04-22 1996-12-17 California Institute Of Technology Ubiquitin-based split protein sensor
EP0781331B1 (en) 1994-08-20 2008-09-03 Gendaq Limited Improvements in or relating to binding proteins for recognition of dna
GB9824544D0 (en) 1998-11-09 1999-01-06 Medical Res Council Screening system
US5789538A (en) 1995-02-03 1998-08-04 Massachusetts Institute Of Technology Zinc finger proteins with high affinity new DNA binding specificities
US5928638A (en) 1996-06-17 1999-07-27 Systemix, Inc. Methods for gene transfer
US5925523A (en) 1996-08-23 1999-07-20 President & Fellows Of Harvard College Intraction trap assay, reagents and uses thereof
GB2338237B (en) 1997-02-18 2001-02-28 Actinova Ltd In vitro peptide or protein expression library
GB9703369D0 (en) 1997-02-18 1997-04-09 Lindqvist Bjorn H Process
US6342345B1 (en) 1997-04-02 2002-01-29 The Board Of Trustees Of The Leland Stanford Junior University Detection of molecular interactions by reporter subunit complementation
GB9710809D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
GB9710807D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
US6410248B1 (en) 1998-01-30 2002-06-25 Massachusetts Institute Of Technology General strategy for selecting high-affinity zinc finger proteins for diverse DNA target sites
ATE466952T1 (de) 1998-03-02 2010-05-15 Massachusetts Inst Technology Poly-zinkfinger-proteine mit verbesserten linkern
US6140081A (en) 1998-10-16 2000-10-31 The Scripps Research Institute Zinc finger binding domains for GNN
US7013219B2 (en) 1999-01-12 2006-03-14 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6503717B2 (en) 1999-12-06 2003-01-07 Sangamo Biosciences, Inc. Methods of using randomized libraries of zinc finger proteins for the identification of gene function
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6599692B1 (en) 1999-09-14 2003-07-29 Sangamo Bioscience, Inc. Functional genomics using zinc finger proteins
US6453242B1 (en) 1999-01-12 2002-09-17 Sangamo Biosciences, Inc. Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites
US7070934B2 (en) 1999-01-12 2006-07-04 Sangamo Biosciences, Inc. Ligand-controlled regulation of endogenous gene expression
US7030215B2 (en) 1999-03-24 2006-04-18 Sangamo Biosciences, Inc. Position dependent recognition of GNN nucleotide triplets by zinc fingers
US6794136B1 (en) 2000-11-20 2004-09-21 Sangamo Biosciences, Inc. Iterative optimization in the design of binding proteins
US20070060606A1 (en) 1999-10-07 2007-03-15 Robertson Harold A Compounds and methods for modulating phosphodiesterase 10A
AU5077401A (en) 2000-02-08 2001-08-20 Sangamo Biosciences Inc Cells for drug discovery
US20020061512A1 (en) 2000-02-18 2002-05-23 Kim Jin-Soo Zinc finger domains and methods of identifying same
CA2407460C (en) 2000-04-28 2013-06-25 Sangamo Biosciences, Inc. Targeted modification of chromatin structure
DE60130645T2 (de) 2000-04-28 2008-01-31 Sangamo BioSciences, Inc., Richmond Datenbanken von regulatorischen sequenzen, methoden zu ihrer herstellung und verwendung
AU2001263155A1 (en) 2000-05-16 2001-11-26 Massachusetts Institute Of Technology Methods and compositions for interaction trap assays
IL154059A0 (en) * 2000-07-21 2003-07-31 Syngenta Participations Ag Zinc finger domain recognition code and uses thereof
JP2002060786A (ja) 2000-08-23 2002-02-26 Kao Corp 硬質表面用殺菌防汚剤
US6919204B2 (en) 2000-09-29 2005-07-19 Sangamo Biosciences, Inc. Modulation of gene expression using localization domains
AU2002217929A1 (en) 2000-11-28 2002-06-11 Sangamo Biosciences, Inc. Modulation of gene expression using insulator binding proteins
US7067317B2 (en) 2000-12-07 2006-06-27 Sangamo Biosciences, Inc. Regulation of angiogenesis with zinc finger proteins
GB0108491D0 (en) 2001-04-04 2001-05-23 Gendaq Ltd Engineering zinc fingers
US20040224385A1 (en) 2001-08-20 2004-11-11 Barbas Carlos F Zinc finger binding domains for cnn
US7262054B2 (en) 2002-01-22 2007-08-28 Sangamo Biosciences, Inc. Zinc finger proteins for DNA binding and gene regulation in plants
CN100575485C (zh) 2002-01-23 2009-12-30 犹他大学研究基金会 使用锌指核酸酶的定向染色体诱变
EP1485475B2 (en) 2002-03-15 2017-09-20 Cellectis Hybrid and single chain meganucleases and use thereof
EP1504092B2 (en) 2002-03-21 2014-06-25 Sangamo BioSciences, Inc. Methods and compositions for using zinc finger endonucleases to enhance homologous recombination
US7074596B2 (en) 2002-03-25 2006-07-11 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Synthesis and use of anti-reverse mRNA cap analogues
US7361635B2 (en) 2002-08-29 2008-04-22 Sangamo Biosciences, Inc. Simultaneous modulation of multiple genes
US9447434B2 (en) 2002-09-05 2016-09-20 California Institute Of Technology Use of chimeric nucleases to stimulate gene targeting
WO2004067736A2 (en) 2003-01-28 2004-08-12 Cellectis Custom-made meganuclease and use thereof
US8409861B2 (en) 2003-08-08 2013-04-02 Sangamo Biosciences, Inc. Targeted deletion of cellular DNA sequences
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
KR100535326B1 (ko) * 2004-01-20 2005-12-09 한국생명공학연구원 줄기 세포로부터 자연살해 세포로의 분화 조절용 유전자를유효성분으로 포함하는 분화 조절제
US7972854B2 (en) 2004-02-05 2011-07-05 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
WO2005099771A2 (en) 2004-04-08 2005-10-27 Sangamo Biosciences, Inc. Methods and compositions for treating neuropathic and neurodegenerative conditions
EP1737498A2 (en) 2004-04-08 2007-01-03 Sangamo Biosciences Inc. Zinc finger proteins for treatment of neuropathic pain
US20080131962A1 (en) 2006-05-25 2008-06-05 Sangamo Biosciences, Inc. Engineered cleavage half-domains
EP1789095A2 (en) 2004-09-16 2007-05-30 Sangamo Biosciences Inc. Compositions and methods for protein production
US7358085B2 (en) * 2005-02-28 2008-04-15 Sangamo Biosciences, Inc. Anti-angiogenic methods and compositions
CA2607104A1 (en) 2005-05-05 2006-11-16 The Arizona Board Of Regents On Behalf Of The University Of Arizona Sequence enabled reassembly (seer) - a novel method for visualizing specific dna sequences
SG10201508995QA (en) 2005-07-26 2015-11-27 Sangamo Biosciences Inc Targeted integration and expression of exogenous nucleic acid sequences
EP2327774A3 (en) 2005-10-18 2011-09-28 Precision Biosciences Rationally-designed meganucleases with altered sequence specificity and DNA-binding affinity
US7320965B2 (en) * 2005-10-28 2008-01-22 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of Huntingtin gene
WO2007136685A2 (en) 2006-05-19 2007-11-29 Sangamo Biosciences, Inc. Methods and compositions for inactivation of dihydrofolate reductase
EP2447279B1 (en) 2006-05-25 2014-04-09 Sangamo BioSciences, Inc. Methods and compositions for gene inactivation
SI2415873T1 (sl) * 2006-12-14 2015-09-30 Dow Agrosciences, Llc Optimizirani nekanonični proteini s cinkovim prstom
EP2415873B1 (en) 2006-12-14 2015-01-21 Dow AgroSciences LLC Optimized non-canonical zinc finger proteins
DE602007005629D1 (de) 2007-06-18 2010-05-12 Commissariat Energie Atomique Reversibles siRNA-Silencing eines mutierten und endogenen Huntington-Wildtypgens und dessen Anwendung zur Behandlung von Morbus Huntington
CN101855231B (zh) 2007-06-19 2014-06-11 路易斯安那州州立大学及农业机械学院管理委员会 信使rna帽的抗-反向硫代磷酸类似物的合成和用途
KR101657504B1 (ko) 2007-09-27 2016-09-19 상가모 바이오사이언스 인코포레이티드 생물학적으로 활성인 뉴클레아제의 신속한 생체내 확인
KR101666228B1 (ko) 2007-09-28 2016-10-13 인트렉손 코포레이션 생물치료학적 분자를 발현시키기 위한 치료학적 유전자-스위치 작제물 및 생물반응기, 및 이의 용도
DE102007056956B4 (de) 2007-11-27 2009-10-29 Moosbauer, Peter, Dipl.-Ing.(FH) Schaltung zur Regelung der Stromversorgung eines Verbrauchers und Verfahren zum Betrieb einer Schaltung
KR101794638B1 (ko) 2008-06-10 2017-12-04 상가모 테라퓨틱스, 인코포레이티드 Ｂａｘ- 및 ｂａｋ-결함 세포주들의 제조 방법 및 조성물
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
US9234016B2 (en) * 2009-07-28 2016-01-12 Sangamo Biosciences, Inc. Engineered zinc finger proteins for treating trinucleotide repeat disorders
US8586526B2 (en) 2010-05-17 2013-11-19 Sangamo Biosciences, Inc. DNA-binding proteins and uses thereof
DK2534173T3 (da) 2010-02-08 2019-10-14 Sangamo Therapeutics Inc Manipulerede spaltnings-halvdomæner
AU2011249019B2 (en) 2010-05-03 2015-01-22 Sangamo Therapeutics, Inc. Compositions for linking zinc finger modules
PL2441774T3 (pl) * 2010-10-15 2019-05-31 Fund Centre De Regulacio Genòmica Peptydy zawierające motywy palców cynkowych i ich zastosowania
US9405700B2 (en) 2010-11-04 2016-08-02 Sonics, Inc. Methods and apparatus for virtualization in an integrated circuit
JP6490426B2 (ja) 2012-02-29 2019-03-27 サンガモ セラピューティクス， インコーポレイテッド ハンチントン病を治療するための方法および組成物

Also Published As

Publication number Publication date
CN108285491A (zh) 2018-07-17
KR20140131973A (ko) 2014-11-14
ZA201406209B (en) 2016-01-27
KR102084539B1 (ko) 2020-03-04
US20130253040A1 (en) 2013-09-26
HK1203566A1 (zh) 2015-10-30
US20200030408A1 (en) 2020-01-30
EP2820159A4 (en) 2015-08-26
US8841260B2 (en) 2014-09-23
US10265377B2 (en) 2019-04-23
JP2019022531A (ja) 2019-02-14
BR112014021104B1 (pt) 2023-03-28
US10857203B2 (en) 2020-12-08
AU2013225950A1 (en) 2014-09-18
BR112014021104A2 (pt) 2017-07-04
US9499597B2 (en) 2016-11-22
CA2865011C (en) 2021-06-15
JP2017143835A (ja) 2017-08-24
JP6490426B2 (ja) 2019-03-27
MX2014010455A (es) 2015-02-10
US20170035839A1 (en) 2017-02-09
US11723952B2 (en) 2023-08-15
US20230414710A1 (en) 2023-12-28
RU2639277C2 (ru) 2017-12-20
EP2820159B1 (en) 2019-10-23
MX374399B (es) 2025-03-06
RU2014138989A (ru) 2016-04-20
AU2013225950B2 (en) 2018-02-15
EP2820159A1 (en) 2015-01-07
CN108285491B (zh) 2021-08-10
CA2865011A1 (en) 2013-09-06
WO2013130824A1 (en) 2013-09-06
US10471123B2 (en) 2019-11-12
JP2015509958A (ja) 2015-04-02
US20210060124A1 (en) 2021-03-04
US20190183970A1 (en) 2019-06-20
NZ629427A (en) 2016-04-29
US20140336133A1 (en) 2014-11-13
CN104204225A (zh) 2014-12-10

Similar Documents

Publication Publication Date Title
MX359327B (es) Composiciones y sus usos para tratar y prevenir la enfermedad de huntington.
PH12019502292A1 (en) Methods and compositions for treatment of a genetic condition
IL267997A (en) Preparations, formulations and methods for treating eye diseases
EP3068905A4 (en) Methods and compositions for treating huntington's disease
EP3139966A4 (en) Methods and compositions for treating huntington's disease
MX2016002544A (es) Compuestos utiles como inmunomoduladores.
WO2014052836A3 (en) Methods and compositions for treating infection
IL236054B (en) Compositions and methods for treating Crohn's disease and related conditions and infections
CR20120202A (es) Métodos y composiciones para tratar cáncer
TR201813877T4 (tr) Spi̇nal müsküler atrofi̇ni̇n tedavi̇ edi̇lmesi̇ne yöneli̇k bi̇leşi̇kler.
NZ628294A (en) Compositions and methods of use of phorbol esters for the treatment of stroke
EP2817018A4 (en) METHODS AND COMPOSITIONS FOR PREVENTING OR TREATING OPHTHALMIC CONDITIONS
DK3292875T3 (en) Compositions and methods for treating diseases
AU2013225816A8 (en) Compositions and methods for treating Type III Gaucher Disease
MY157341A (en) Methods and compositions for treating lung cancer
EP4289820A3 (en) Compounds for use in treating alzheimer's disease in apoe4+/+ patients
IN2014KN02620A (es)
WO2013023151A3 (en) Compositions and methods for treating celiac sprue disease
MX359181B (es) Hidrolizado de colágeno y uso del mismo.
GB201302343D0 (en) Pharmaceutical compositions comprising 15-OHEPA and methods for using the same
MX367393B (es) Composiciones antifungicas para el tratamiento de piel y uñas.
MX2013014488A (es) Metodos y composiciones para tratar el cancer cerebral.
EP2542057A4 (en) COMPOSITIONS AND METHODS FOR TREATING OR PREVENTING INFAMOUS DISEASES
EP3041485A4 (en) Cell-seeded compositions and methods useful for treating bone regions
TN2010000463A1 (en) Compositions and methods for preparing and using same

Legal Events

Date Code Title Description
FG Grant or registration