A Phase I Clinical Trial of AAV2‐BDNF Gene Therapy for Alzheimer's Disease: Findings

AAV2-BDNF gene therapy to the entorhinal cortex in mild AD has been safe to date and restores FDG-PET activity in the treated brain region. BDNF gene therapy offers the potential to prevent neuronal loss, activate neuronal function and restore synapses, representing a possible neuro-restorative treatment for AD and MCI.

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Publication Details

Journal
Alzheimer s & Dementia
Published
2025-12-01
DOI
https://doi.org/10.1002/alz70859_101305
Primary Topic
Nerve injury and regeneration
Type
article
Field-Weighted Citation Impact
0.00
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article

A Phase I Clinical Trial of AAV2‐BDNF Gene Therapy for Alzheimer's Disease: Findings

Russell R. Lonser, M H Tuszynski, Gabriel C. Léger, Douglas W. Scharre et al.
Alzheimer s & Dementia
Nerve injury and regeneration
article

A Phase I Clinical Trial of AAV2‐BDNF Gene Therapy for Alzheimer's Disease: Findings

Russell R. Lonser, M H Tuszynski, Gabriel C. Léger, Douglas W. Scharre, Krystof S Bankiewicz, J. Bradley Elder
article en

Abstract

AAV2-BDNF gene therapy to the entorhinal cortex in mild AD has been safe to date and restores FDG-PET activity in the treated brain region. BDNF gene therapy offers the potential to prevent neuronal loss, activate neuronal function and restore synapses, representing a possible neuro-restorative treatment for AD and MCI.

Alzheimer s & DementiaVol. 21(S5)
United States Department of Veterans Affairs (US), The Ohio State University Wexner Medical Center (US), University of California San Diego (US), The Ohio State University (US)
Quality Education
Openalex Percentile: Top 37%
Nerve injury and regeneration
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