Developing targeted gene therapies for rare genetic brain diseases
Engineered AAV vectors for combinatorial treatment of rare genetic brain diseases
This study is working on new ways to deliver gene therapy specifically for rare brain disorders caused by harmful genes, like Spinocerebellar Ataxia Type 7, to help improve treatment options for patients.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California-Irvine NIH-funded |
| Lab location | 1 site (Irvine, United States) |
| Project ID | NIH-10694918 on NIH RePORTER |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with rare genetic brain disorders, particularly those caused by toxic gene products.
Not a fit: Patients with common neurological disorders or those not affected by genetic brain diseases may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could provide new, effective treatment options for patients suffering from rare genetic brain diseases.
How similar studies have performed: Other research has shown promise in using AAV vectors for gene therapy, indicating a potential for success with this innovative approach.
Where this research is happening
Irvine, United States
- University of California-Irvine — Irvine, United States (Active)
Researchers
- Principal investigator: Xu, Xiangmin — University of California-Irvine
- Study coordinator: Xu, Xiangmin
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.