Developing targeted gene therapies for rare genetic brain diseases

Who could benefit from this research

What this research studies

This research focuses on creating advanced AAV vectors for gene therapy aimed at treating rare genetic brain disorders caused by toxic gene products. The team will design and manufacture new vectors that enhance safety, efficacy, and target specificity for delivering therapeutic genes to neurons. By utilizing innovative techniques such as neuron-specific delivery and temporal control of gene expression, the project aims to improve treatment options for conditions like Spinocerebellar Ataxia Type 7 and VCP multisystem proteinopathy. The research will also employ advanced analytics to assess the effectiveness of these gene therapies in mouse models.

Why it matters

Where this research is happening

• University of California-Irvine
  Irvine, United States Active

Researchers

• Principal investigator: Xu, Xiangmin — University of California-Irvine
• Study coordinator: Xu, Xiangmin

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About this research

1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
3. For full project details, budget, and progress reports, visit the official NIH RePORTER page.