Adeno-associated virus (AAV) is a small, nonenveloped virus that was adapted 30 years
ago for use as a gene transfer vehicle. It is capable of transducing a wide range of species …

Inherited retinopathies (IR) are common untreatable blinding conditions. Most of them are
inherited as monogenic disorders, due to mutations in genes expressed in retinal …

Gene transfer is an increasingly utilized approach for research and clinical applications
involving the central nervous system (CNS). Vectors for gene transfer can be as simple as …

Abstract Usher syndrome 1B (USH1B) is a severe, autosomal recessive, deaf–blind disorder
caused by mutations in myosin 7A (MYO7A). Patients are born profoundly deaf and exhibit …

From Bench to Bedside''was held June 15 and 16, 2012, at the University of Colorado
Anschutz Medical Campus in Aurora, Colorado. The SERC provided a diverse group of …

Recombinant adeno-associated viruses are important vectors for retinal gene delivery.
Currently utilized vectors have relatively slow onset, and for efficient transduction it is …

Retinal degeneration, including that seen in age-related macular degeneration and retinitis
pigmentosa (RP), is the most common form of neural degenerative disease in the world …

Adeno-associated virus (AAV)-mediated gene delivery has emerged as an effective and
safe tool for both preclinical and clinical studies of neurological disorders. The recent …

Adeno-associated virus (AAV) is a small, non-pathogenic dependovirus that has shown
great potential for safe and long-term expression of a genetic payload in the retina. AAV has …

Despite their physiological roles, Müller glial cells are involved directly or indirectly in retinal
disease pathogenesis and are an interesting target for therapeutic approaches for retinal …