AAV-BDNF-WPRE

AAV-BDNF-WPRE is a gene therapy utilizing an adeno-associated virus (AAV) vector engineered to deliver and express human brain-derived neurotrophic factor (BDNF), often with a woodchuck hepatitis virus posttranscriptional regulatory element (WPRE) to enhance transgene expression. The therapy is designed for central nervous system delivery, typically intracerebral. BDNF acts as a neurotrophic factor supporting neuronal survival, synaptic plasticity, remyelination, metabolic regulation, and energy homeostasis. Mechanistically, BDNF increases expression and signaling through its primary receptor tropomyosin receptor kinase B (TrkB), activating downstream neuroprotective and metabolic pathways. Preclinical studies show efficacy in models of Prader-Willi syndrome (obesity, metabolic dysfunction) and demyelinating disease (multiple sclerosis), with improved cognition, metabolism, insulin sensitivity, and remyelination. Clinical trials in early Alzheimer's disease and mild cognitive impairment have begun.

Other names

adeno-associated virus expressing brain-derived neurotrophic factorAAV2-BDNFAAV-2-BDNFAAV 2-BDNFAAV-BDNF gene therapy