Evidence Hub

Adeno-associated virus-mediated neural repair in parkinson's disease: a systematic review from animal models to clinical trials

Yancheng Wang

Journal of Clinical Technology and Theory, 2025

Abstract

In response to the limitations of existing treatment methods, the development of new treatment approaches that can delay or reverse the neurodegenerative process has become a research hotspot. Gene therapy, especially strategies based on adeno-associated virus (AAV) vectors, is emerging as a research frontier in the treatment of Parkinson's disease due to its high efficiency, low immunogenicity, long-term stable expression, and neuron tropism in the nervous system. Recent clinical trials have demonstrated significant improvements in motor function, with some approaches showing up to 36% enhancement in UPDRS scores. AAV-mediated gene therapy mainly focuses on the following therapeutic targets: dopamine pathway reconstruction, neurotrophic factor delivery, -synuclein clearance strategies, and emerging therapeutic targets. The main purpose of this review is to evaluate the latest research progress of AAV vector gene therapy in Parkinson's disease, focusing on analyzing its potential and challenges in the preclinical and clinical translation process and proposing possible optimization paths to promote the clinical application of gene therapy.

How strong is the evidence?

Evidence Hub reads this as a systematic review, done in people. Its evidence level is 1 of 5 (Highest · pooled evidence). Pooled evidence from several studies sits at the top of the evidence pyramid.

How the five levels of evidence work. The level is assigned automatically from the abstract and can be wrong, so check the paper.

Read the paper

Related studies

Go deeper in Evidence Hub

Evidence Hub adds a study data sheet (design, sample size, evidence level, key numbers), an AI summary and a chat grounded in the paper's text.

Open this study Sign up free