Potential Gene Therapy TSHA-102 Does Well in Animal Study

Potential Gene Therapy TSHA-102 Does Well in Animal Study

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Potential Gene Therapy TSHA-102 Does Well in Animal Study

TSHA-102, an experimental gene therapy for Rett syndrome, safely and effectively normalized levels of MeCP2 — the protein missing or defective in most Rett patients — in the brain and significantly improved survival in a mouse model of the disease, a study shows. Notably, the data suggest this investigational treatment can overcome a common issue of MECP2-directed gene therapy strategies: excessive levels of MeCP2, which are toxic to cells. “It has been a challenge finding an approach that…

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