Inactive

Over the last several decades, human gene therapy (GT) has significantly advanced such that thousands of clinical trials are being tested worldwide and hundreds of those have used adeno-associated vir... Over the last several decades, human gene therapy (GT) has significantly advanced such that thousands of clinical trials are being tested worldwide and hundreds of those have used adeno-associated virus (AAV) vectors. While viral vectors could be used for the treatment of multiple diseases with abnormalities in the same cell types, the current approach to gene therapy clinical trials is still “one disease at a time,” which limits the potential capacity of viral vectors as a broad therapeutic platform. The Neuromuscular and Neurogenetic Disorders of Childhood Section (NNDCS) of the National Institute of Neurological Disorders and Stroke (NINDS) is working on a platform GT approach to target the neuromuscular junction (NMJ). The NINDS will be performing electrophysiology intracellular recordings measurements on mouse tissue as part of this study, which will be an important outcome measure for measuring NMJ in the NINDS’ preclinical models. Please see the attached source sought notice for more information.