Adeno-associated viral (AAV) vectors are a rapidly emerging gene therapy platform for the
treatment of neurological diseases. In preclinical studies, transgenes encoding therapeutic …

Astrocytes are a type of glial cell that tile the CNS. They interact with multiple cell types,
including neurons, glial cells and blood vessels, and are involved or implicated in brain …

Both observational and perturbational technologies are essential for advancing the
understanding of brain function and dysfunction. But while observational techniques have …

Gene therapy is at the cusp of a revolution for treating a large spectrum of CNS disorders by
providing a durable therapeutic protein via a single administration. Adeno-associated virus …

Currently there is no general approach for achieving specific optogenetic control of
genetically defined cell types in rats, which provide a powerful experimental system for …

Mice lacking the vesicular glutamate transporter-3 (VGLUT3) are congenitally deaf due to
loss of glutamate release at the inner hair cell afferent synapse. Cochlear delivery of …

Recombinant viruses allow for targeted transgene expression in specific cell populations
throughout the nervous system. The adeno-associated virus (AAV) is among the most …

Noninvasive systemic gene delivery to the central nervous system (CNS) has largely been
impeded by the blood–brain barrier (BBB). Recent studies documented widespread CNS …

The potential benefits of gene therapy for neurological diseases such as Parkinson's,
Amyotrophic Lateral Sclerosis (ALS), Epilepsy, and Alzheimer's are enormous. Even a delay …

The non-pathogenic parvovirus, adeno-associated virus (AAV), is an efficient vector for
transgene expression in vivo and shows promise for treatment of brain disorders in clinical …