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 …

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 …

Background Alzheimer's disease (AD) is a progressive and age-associated
neurodegenerative disorder that affects women disproportionally. However, the underlying …

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

In recent years, gene therapy has been raising hopes toward viable treatment strategies for
rare genetic diseases for which there has been almost exclusively supportive treatment. We …

Late infantile neuronal ceroid lipofuscinosis (LINCL) is an autosomal recessive,
neurodegenerative lysosomal storage disease affecting the CNS and is fatal by age 8 to 12 …

Congenital neuronal ceroid-lipofuscinosis (NCL) is a devastating inherited
neurodegenerative disorder of unknown metabolic basis. Eight patients with this rare …

The neuronal ceroid lipofuscinoses (NCL) are severe neurodegenerative lysosomal storage
disorders of childhood, characterized by accumulation of autofluorescent ceroid …

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