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 …

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

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 …

The recent market approvals of recombinant adeno-associated virus (rAAV) gene therapies
in Europe and the United States are landmark achievements in the history of modern …

Retinitis Pigmentosa (RP) is a hereditary retinopathy that affects about 2.5 million people
worldwide. It is characterized with progressive loss of rods and cones and causes severe …

Gene therapy has historically been defined as the addition of new genes to human cells.
However, the recent advent of genome-editing technologies has enabled a new paradigm in …

The mammalian retina provides an excellent opportunity to study glia–neuron interactions
and the interactions of glia with blood vessels. Three main types of glial cells are found in …

Background Mutations in RPE65 cause Leber's congenital amaurosis, a progressive retinal
degenerative disease that severely impairs sight in children. Gene therapy can result in …