Hereditary diseases are caused by mutations in genes, and more than 7,000 rare diseases
affect over 30 million Americans. For more than 30 years, hundreds of researchers have …

Adeno-associated virus (AAV) vectors are the leading platform for gene delivery for the
treatment of a variety of human diseases. Recent advances in develo** clinically …

Gene therapy with adeno-associated virus (AAV) vectors has demonstrated safety and long-
term efficacy in a number of trials across target organs, including eye, liver, skeletal muscle …

Adeno-associated virus (AAV) has emerged as a leading platform for gene delivery for
treating various diseases due to its excellent safety profile and efficient transduction to …

Cancer is difficult to cure due to frequent metastasis, and develo** effective therapeutic
approaches to treat cancer is urgently important. Long non-coding RNAs (lncRNAs) have …

BACKGROUND Nearly five decades ago, visionary scientists hypothesized that genetic
modification by exogenous DNA might be an effective treatment for inherited human …

Recombinant adeno-associated viruses (AAVs) have emerged as promising gene delivery
vehicles resulting in three US Food and Drug Administration (FDA) and one European …

Background Phase 1 studies have shown potential benefit of gene replacement in RPE65-
mediated inherited retinal dystrophy. This phase 3 study assessed the efficacy and safety of …

CEP290-associated Leber congenital amaurosis type 10 (LCA10) is a retinal disease
resulting in childhood blindness. Sepofarsen is an RNA antisense oligonucleotide targeting …

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 …