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AAV2-BDNF is an investigational gene therapy designed to deliver the human brain-derived neurotrophic factor (BDNF) gene directly into targeted regions of the brain using an adeno-associated virus serotype 2 (AAV2) vector. BDNF is a neurotrophic growth factor that supports neuronal survival, stimulates cell function, and promotes synaptic connectivity in key memory circuits such as the entorhinal cortex and hippocampus. The therapy aims to slow or prevent neuronal loss and potentially restore function in patients with early Alzheimer's disease (AD) or mild cognitive impairment (MCI). Because BDNF does not cross the blood-brain barrier, direct intraparenchymal injection of the viral vector is used to achieve localized expression of BDNF within affected brain regions. This approach seeks to rebuild neural circuits, slow cell loss, and stimulate remaining neurons[1][3][5][7][8].
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