University of Rochester School of Medicine and Dentistry Researcher Targets Gene Therapy (Ribozyme-activated mRNA trans-ligation enables large gene delivery to treat muscular dystrophies).

Předmět:	NEUROLOGICAL disorders MUSCULAR dystrophy NEUROMUSCULAR diseases CATALYTIC RNA MUSCULOSKELETAL system diseases
Zdroj:	Gene Therapy Weekly; 12/6/2024, p4345-4345, 1p
Abstrakt:	Researchers at the University of Rochester School of Medicine and Dentistry have developed a new gene therapy technique called StitchR, which involves ribozyme-activated mRNA trans-ligation to deliver large genes for treating muscular dystrophies. This innovative approach resulted in a significant increase in protein expression in mammalian cells, approaching levels seen with single vectors. The study demonstrates the potential of StitchR for dual adeno-associated virus gene therapies to correct muscular dystrophies by restoring large functional muscle proteins to normal levels in vivo. [Extracted from the article]
Databáze:	Complementary Index
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