Over the past decade, pre-clinical and clinical trials have demonstrated that gene therapy is a viable precision medicine approach for treating mitochondrial diseases. However, several challenges remain, including vector design, targeted tissue tropism and efficient delivery, transgene expression, and immunotoxicity. Two recent articles overviewed the basics of mitochondrial diseases and clinical research for personalized mitochondrial medicine.
- Suomalainen A, Nunnari J. Mitochondria at the crossroads of health and disease. Cell. 2024 May 23;187(11):2601-27.
- Gropman AL, Uittenbogaard MN, Chiaramello AE. Challenges and opportunities to bridge translational to clinical research for personalized mitochondrial medicine. Neurotherapeutics. 2024 Jan 19:e00311.
A discussion on mitochondrial gene therapy would be greatly appreciated.
Best regards.
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Sudip Das, MPharm, PhD
Distinguished Faculty, Butler University
Professor of Pharmaceutics & Drug Delivery
4600 Sunset Avenue, Indianapolis, IN 46208-3485
E-mail:
[email protected]Webpage:
https://research.butler.edu/nanomedicine/LinkedIn:
http://www.linkedin.com/in/sudipkdasDisclaimer: Opinions expressed are solely my own and do not express the views or opinions of my employer.
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