To date, almost 2600 gene therapy clinical trials have been completed, are ongoing or have
been approved worldwide. Our database brings together global information on gene therapy …

Duchenne muscular dystrophy (DMD) has been a major target for gene therapy
development for nearly 30 years. DMD is among the most common genetic diseases, and …

Gene replacement therapies utilizing adeno-associated viral (AAV) vectors hold great
promise for treating Duchenne muscular dystrophy (DMD). A related approach uses AAV …

Muscular dystrophies are a heterogeneous group of genetic diseases, characterized by
progressive degeneration of skeletal and cardiac muscle. Despite the intense investigation …

Usher syndrome type 1 F (USH1F), caused by mutations in the protocadherin-15 gene
(PCDH15), is characterized by congenital deafness, lack of balance, and progressive …

Neurological disorders–disorders of the brain, spine and associated nerves–are a leading
contributor to global disease burden with a shockingly large associated economic cost …

Clustered regularly interspaced short palindromic repeats (CRISPR) is a promising
innovative technology for genomic editing that offers scientists the chance to edit DNA …

Pediatric neuromuscular diseases encompass all disorders with onset in childhood and
where the primary area of pathology is in the peripheral nervous system. These conditions …

Micro-dystrophins are highly promising candidates for treating Duchenne muscular
dystrophy, a lethal muscle disease caused by dystrophin deficiency. Here, we report robust …

Duchenne muscular dystrophy (DMD) is one of the most devastating neuromuscular genetic
diseases caused by the absence of dystrophin. The continuous episodes of muscle …