Overview

• 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.