Recombinant adeno-associated virus vectors (rAAV) are being explored as gene delivery
vehicles for the treatment of various inherited and acquired disorders. rAAVs are attractive …

The use of adeno-associated viral (AAV) vectors for gene therapy treatments of inherited
disorders has accelerated over the past decade with multiple clinical trials ongoing in …

Autosomal recessive genetic forms (DFNB) account for most cases of profound congenital
deafness. Adeno-associated virus (AAV)-based gene therapy is a promising therapeutic …

Inherited retinal dystrophies and optic neuropathies cause chronic disabling loss of visual
function. The development of recombinant adeno-associated viral vectors (rAAV) gene …

Gene therapy with adeno-associated viral (AAV) vectors has reached the clinical stage for
many inherited and acquired diseases. However, due to a cargo capacity limited to< 5 kb …

Deficiency of the dysferlin protein presents as two major clinical phenotypes: limb–girdle
muscular dystrophy type 2B and Miyoshi myopathy. Dysferlin is known to participate in …

The trans-splicing (ts) and overlapping (ov) vectors expand the packaging capacity of adeno-
associated virus (AAV). But their application depends on the inherent properties of the target …

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 …

Gene delivery using recombinant adeno-associated virus (rAAV) has emerged to the
forefront demonstrating safe and effective phenotypic correction of diverse diseases …

Gene therapy using adeno-associated viral (AAV) vectors currently represents the most
promising approach for the treatment of many inherited retinal diseases (IRDs), given AAV's …