Applied Genetic Technologies Corporation
ADENO-ASSOCIATED VIRUS (AAV) SYSTEMS FOR TREATMENT OF PROGRANULIN ASSOCIATED NEURODEGENERATIVE DISEASES OR DISORDERS

Abstract:

The disclosure provides, in part, optimally-modified progranulin (PGRN) cDNA and associated genetic elements for use in recombinant adeno-associated virus (rAAV)-based gene therapy for neurodegenerative disorders characterized by cognitive disruption, behavioral impairment, and deficient lysosomal storage, including familial frontotemporal dementia (FTD), frontotemporal lobar degeneration (FTLD), neuronal ceroid lipofuscinosis (NCL), or Alzheimer's disease (AD).