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CY1124197T1 - Antinohmatika πολυνουκλεοτιδια για να διεγειρεται παραλειψη εξονιου και μεθοδοι θεραπειας δυστροφιων - Google Patents

Antinohmatika πολυνουκλεοτιδια για να διεγειρεται παραλειψη εξονιου και μεθοδοι θεραπειας δυστροφιων

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Publication number
CY1124197T1
CY1124197T1 CY20211100476T CY211100476T CY1124197T1 CY 1124197 T1 CY1124197 T1 CY 1124197T1 CY 20211100476 T CY20211100476 T CY 20211100476T CY 211100476 T CY211100476 T CY 211100476T CY 1124197 T1 CY1124197 T1 CY 1124197T1
Authority
CY
Cyprus
Prior art keywords
exon
seq
methods
polynucleotides
polynucleotide
Prior art date
Application number
CY20211100476T
Other languages
English (en)
Inventor
Elizabeth MCNALLY
Original Assignee
The University Of Chicago
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 The University Of Chicago filed Critical The University Of Chicago
Publication of CY1124197T1 publication Critical patent/CY1124197T1/el

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    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1138—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against receptors or cell surface proteins
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Abstract

Παρέχονται αντινοηματικά πολυνουκλεοτίδια και χρήση αυτών σε φαρμακευτικές συνθέσεις για να διεγείρεται παράλειψη εξονίου σε στοχευόμενα εξόνια του γονιδίου γάμμα σαρκογλυκάνης, μαζί με μεθόδους αποτροπής ή θεραπείας δυστροφικών ασθενειών, όπως Ζωνιαίας Μυϊκής Δυστροφίας. Μία άποψη της αποκάλυψης παρέχει απομονωθέν αντινοηματικό πολυνουκλεοτίδιο, όπου το πολυνουκλεοτίδιο ειδικώς υβριδοποιεί προς περιοχή εξονίου στόχου ενός RNA γάμμα σαρκογλυκάνης, όπου το εξόνιο επιλέγεται από την ομάδα που αποτελείται από εξόνιο 4 (SEQ ID ΝΟ;1), εξόνιο 5 (SEQ ID NO: 2), εξόνιο 6 (SEQ ID NO: 3), εξόνιο 7 (SEQ ID NO: 4) και συνδυασμούς αυτών. Σε μερικές πραγματοποιήσεις το αντινοηματικό πολυνουκλεοτίδιο δεν μπορεί να σχηματίσει υπόστρωμα RΝάσης Η και σε περαιτέρω πραγματοποιήσεις το αντινοηματικό πολυνουκλεοτίδιο περιλαμβάνει ραχοκοκαλιά τροποποιημένου πολυνουκλεοτιδίου.
CY20211100476T 2012-09-06 2021-06-01 Antinohmatika πολυνουκλεοτιδια για να διεγειρεται παραλειψη εξονιου και μεθοδοι θεραπειας δυστροφιων CY1124197T1 (el)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261697766P 2012-09-06 2012-09-06
PCT/US2013/058636 WO2014039916A1 (en) 2012-09-06 2013-09-06 Antisense polynucleotides to induce exon skipping and methods of treating dystrophies
EP13835266.1A EP2892617B1 (en) 2012-09-06 2013-09-06 Antisense polynucleotides to induce exon skipping and methods of treating dystrophies

Publications (1)

Publication Number Publication Date
CY1124197T1 true CY1124197T1 (el) 2022-05-27

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CY181100911T CY1121037T1 (el) 2012-09-06 2018-09-03 Αντινοηματικα πολυνουκλεοτιδια για να διεγειρεται παραλειψη εξονιου και μεθοδοι θεραπειας δυστροφιων
CY20211100476T CY1124197T1 (el) 2012-09-06 2021-06-01 Antinohmatika πολυνουκλεοτιδια για να διεγειρεται παραλειψη εξονιου και μεθοδοι θεραπειας δυστροφιων

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US (13) US9499817B2 (el)
EP (2) EP2892617B1 (el)
BR (1) BR112015005091B1 (el)
CA (1) CA2884245C (el)
CY (2) CY1121037T1 (el)
DK (2) DK3421602T3 (el)
ES (2) ES2686727T3 (el)
HK (1) HK1212272A1 (el)
HR (2) HRP20181458T1 (el)
HU (2) HUE039676T2 (el)
LT (1) LT3421602T (el)
PL (1) PL3421602T3 (el)
PT (2) PT3421602T (el)
RS (2) RS61985B1 (el)
SI (2) SI3421602T1 (el)
TN (1) TN2015000079A1 (el)
WO (1) WO2014039916A1 (el)

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US20240076670A1 (en) 2024-03-07
EP2892617B1 (en) 2018-06-13
US20240327844A1 (en) 2024-10-03
US9499817B2 (en) 2016-11-22
LT3421602T (lt) 2021-05-25
EP2892617A1 (en) 2015-07-15
PT3421602T (pt) 2021-05-31
US20210403910A1 (en) 2021-12-30
HUE054260T2 (hu) 2021-08-30
US20200040340A1 (en) 2020-02-06
US20230183700A1 (en) 2023-06-15
US20150225718A1 (en) 2015-08-13
US9777271B2 (en) 2017-10-03
ES2686727T3 (es) 2018-10-19
EP3421602B1 (en) 2021-03-03
ES2878650T3 (es) 2021-11-19
WO2014039916A1 (en) 2014-03-13
CA2884245C (en) 2023-03-14
US20210147845A1 (en) 2021-05-20
RS61985B1 (sr) 2021-07-30
RS57789B1 (sr) 2018-12-31
US20180037889A1 (en) 2018-02-08
TN2015000079A1 (en) 2016-06-29
BR112015005091B1 (pt) 2021-07-06
HRP20210877T1 (hr) 2021-07-23
EP3421602A1 (en) 2019-01-02
US20160369277A1 (en) 2016-12-22
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EP2892617A4 (en) 2016-06-22
SI3421602T1 (sl) 2021-08-31
US20200291403A1 (en) 2020-09-17
BR112015005091A2 (pt) 2017-09-26
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CY1121037T1 (el) 2019-12-11
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