Adeno-associated virus (AAV) vectors can deliver transgenes to diverse cell types and are
therefore useful for basic research and gene therapy. Although AAV has many advantages …

Highlights•Only intravascular delivery can truly change the course of systemic
diseases.•AAV has the capacity to escape from the blood and lead to bodywide gene …

For dominantly inherited disorders development of gene therapies, targeting the primary
genetic lesion has been impeded by mutational heterogeneity. An example is rhodopsin …

Inverted terminal repeats (ITRs) are the only wild-type components retained in the genome
of adeno-associated virus (AAV) vectors. To determine whether ITR modification is a viable …

We describe biophysical and ultrastructural differences in genome release from adeno-
associated virus (AAV) capsids packaging wild-type DNA, recombinant single-stranded DNA …

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 …

Objective Dysferlinopathies are a family of untreatable muscle disorders caused by
mutations in the dysferlin gene. Lack of dysferlin protein results in progressive dystrophy …

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

Over the last few years, huge progress has been made with regard to the understanding of
molecular mechanisms underlying the pathogenesis of neurodegenerative diseases of the …