Gene therapy is a promising approach for treating a spectrum of neurological and
neurodegenerative disorders by delivering corrective genes to the central nervous system …

Gene therapy has strong potential for treating a variety of genetic disorders, as
demonstrated in recent clinical trials. There is unfortunately no scarcity of disease targets …

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 …

The past several years have witnessed significant advances in the development of
therapeutic gene delivery for neurological disorders of the central nervous system (CNS). In …

Adeno-associated viral vectors (AAVs) are increasingly useful preclinical tools in
neuroscience research studies for interrogating cellular and neurocircuit functions and …

Adeno-associated virus (AAV)-mediated gene delivery has emerged as an effective and
safe tool for both preclinical and clinical studies of neurological disorders. The recent …

Diseases of the central nervous system (CNS) have provided enormous opportunities for the
therapeutic application of viral vector gene transfer. Adeno-associated virus (AAV) has been …

Gene therapy is a powerful tool to treat various central nervous system (CNS) diseases
ranging from monogenetic diseases to neurodegenerative disorders. Adeno-associated …

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 …

Gene delivery to, and gene targeting in, stem cells would be a highly enabling technology
for basic science and biomedical application. Adeno-associated viral (AAV) vectors have …