MicroRNAs (miRNAs) are small non-coding RNAs crucial for post-transcriptional and
translational regulation of cellular and developmental pathways. The study of miRNAs in …

β-Thalassemia is brought about by defective β-globin (HBB [hemoglobin subunit β]) formation
and, in severe cases, requires regular blood transfusion and iron chelation for survival. …

Diamond-Blackfan anemia syndrome (DBAS) is a rare inherited bone marrow failure (BMF)
syndrome characterized by erythroid aplasia, congenital malformations, and cancer …

Molecular therapies and functional studies greatly benefit from spatial and temporal precision
of genetic intervention. We therefore conceived and explored tag-activated microRNA (…

Introduction: Genome editing tools, such as CRISPR/Cas, TALE nucleases and, more recently,
double-strand-break-independent editors, have been successfully used for gene therapy …

Background and Objective The standard of care in Acute Myeloid Leukemia patients has
remained essentially unchanged for nearly 40 years. Due to the complicated mutational …

Therapy via the gene addition of the anti-sickling β AS3 -globin transgene is potentially curative
for all β-hemoglobinopathies and therefore of particular clinical and commercial interest. …

Advanced therapy medicinal products (ATMPs) are medicines for human use based on genes,
cells or tissue engineering. After clear successes in adults, the nascent technology now …

Cellular and molecular immune components play a crucial role in the development and
perpetuation of human malignancies, shaping anti-tumor responses. A novel immune regulator …

Background and Objective: The standard of care in Acute Myeloid Leukemia patients has
remained essentially unchanged for nearly 40 years. Due to the complicated mutational …