Achieving sufficient delivery across the blood–brain barrier is a key challenge in the
development of drugs to treat central nervous system (CNS) disorders. This is particularly …

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 developing 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 …

Recombinant adeno-associated viruses (AAVs) are commonly used gene delivery vehicles
for neuroscience research. They have two engineerable features: the capsid (outer protein …

There has been a resurgence in gene therapy efforts that is partly fueled by the identification
and understanding of new gene delivery vectors. Adeno-associated virus (AAV) is a non …

Neurotropic adeno-associated virus (AAV) serotypes such as AAV9 have been
demonstrated to transduce spinal alpha motor neurons when administered intravenously (iv) …

Alzheimer's disease (AD) and treatment of the brain in aging require the development of
new biologic drugs, such as recombinant proteins or gene therapies. Biologics are large …

Gene transfer has long been a compelling yet elusive therapeutic modality. First mainly
considered for rare inherited disorders, gene therapy may open treatment opportunities for …

Crossing the blood–brain barrier in primates is a major obstacle for gene delivery to the
brain. Adeno-associated viruses (AAVs) promise robust, non-invasive gene delivery from the …