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

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 therapy is rapidly emerging as a powerful therapeutic strategy for a wide range of
neurodegenerative disorders, including Alzheimer's disease (AD), Parkinson's disease (PD) …

Viral vectors are a promising tool for effective delivery of genetic material into cells. They
take advantage of the natural ability of a virus to deliver a genetic payload into cells while …

Batten disease (also known as neuronal ceroid lipofuscinoses) constitutes a family of
devastating lysosomal storage disorders that collectively represent the most common …

Abstract Lysosomal Storage Disorders (LSDs) are a diverse group of inherited, monogenic
diseases caused by functional defects in specific lysosomal proteins. The lysosome is a …

The neuronal ceroid lipofuscinoses comprise a group of neurodegenerative lysosomal
storage disorders caused by mutations in at least 13 different genes and primarily affect the …

Loss-of-function mutations in progranulin, a lysosomal glycoprotein, cause
neurodegenerative disease. Progranulin haploinsufficiency causes frontotemporal dementia …

Alzheimer's disease (AD) is a progressive degenerative neurological disorder affecting
nearly one in nine elderly people in the United States. Population studies have shown that …

Global gene delivery to the CNS has therapeutic importance for the treatment of
neurological disorders that affect the entire CNS. Due to direct contact with the CNS …