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TAFAZZIN gene replacement therapy Background Barth Syndrome (BTHS) is an X-linked, potentially lethal genetic disease that affects about 1 in 0.3 to 0.4 million live births. It is characterized by cardiomyopathy, skeletal myopathy, neutropenia, growth delay, poor feeding, and organic aciduria, with cardiac complications and neutropenia being the...

Published: 3/30/2026 | Updated: 3/10/2025 | Inventor(s): William Pu, Suya Wang

Keywords(s):

Category(s): Technology Classifications > Disease Area > Rare Diseases, Technology Classifications > Invention Type > Therapeutics > Gene Therapy, Technology Classifications > Disease Area > Cardiology

In vivo high efficiency somatic homology directed repair

In vivo high-efficiency somatic homology directed repair Background Recent advances in genome sequencing techniques and analysis methods have significantly accelerated the ability to catalog and map genetic factors associated with a diverse range of biological functions and diseases, but precise genome targeting technologies are needed to enable...

Published: 3/30/2026 | Updated: 2/23/2024 | Inventor(s): William Pu, Nathan VanDusen, Yanjiang Zheng

Keywords(s):

Category(s): Technology Classifications > Disease Area > Cardiology, Technology Classifications > Invention Type > Therapeutics > Gene Therapy