New Gene Editing Tool Targets Multiple Diseases Simultaneously
Researchers at the University of Pennsylvania develop mvGPT, a groundbreaking platform combining gene editing and expression regulation in a single tool.
- The mvGPT tool can edit DNA, activate gene expression, and repress gene expression simultaneously, addressing multiple genetic conditions at once.
- This innovative technology combines an improved 'Prime Editor' with mechanisms to upregulate and downregulate genes independently and precisely.
- Initial tests on human liver cells successfully corrected a mutation causing Wilson's disease while targeting genes linked to type I diabetes and transthyretin amyloidosis.
- The compact design of mvGPT simplifies delivery into cells, using methods like mRNA strands or viral vectors, reducing complexity in genetic therapies.
- Future research will test mvGPT in animal models and explore its potential to treat other genetic diseases, including cardiovascular conditions.
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