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Gene therapy for alcohol-use disorder

We performed gene therapy to induce expression of GDNF in the ventral tegmental area of rhesus monkey brains. Our approach countered the hypodopaminergic state that is associated with chronic alcohol drinking and reduced alcohol intake to fewer than two drinks per day while other ingestive behaviors remained intact. These findings, along with results from our ongoing clinical trials of GDNF gene therapy in other diseases, support future application of this approach in humans with alcohol-use disorder.

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Fig. 1: MR-guided delivery of AAV2-hGDNF to the brain reduces alcohol consumption in a rhesus macaque model of AUD.


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This is a summary of: Ford, M. M. et al. GDNF gene therapy for alcohol use disorder in male non-human primates. Nat. Med. (2023).

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Gene therapy for alcohol-use disorder. Nat Med 29, 1914–1915 (2023).

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