Gene-Modified Stem Cell Therapy for Subjects With Transfusion-dependent Beta-thalassemia

Gene therapy restored hearing in deaf patients

CRISPR gene therapy insights may improve access to sickle cell cures

Gene therapy for children with sickle cell disease approved by FDA

Non-genotoxic transplantation and in vivo selection through epitope editing

FDA Approves First Dual-AAV Gene Therapy for Genetic Hearing Loss Under Priority Voucher Program

The FDA has approved Otarmeni (lunsotogene parvec-cwha), the first-ever dual adeno-associated virus (AAV) vector-based gene therapy for trea

Hematopoietic stem cell gene therapy delivers cell-penetrating frataxin to treat Friedreich's ataxia in preclinical models

This article describes a novel gene and cell therapy approach for Friedreich's ataxia (FRDA), a neurodegenerative disease caused by frataxin