Foundation for Prader-Willi Blog | Genetic Therapy

Exciting new research funded by FPWR has brought us one step closer to an effective gene therapy for Prader-Willi syndrome (PWS). Led by Dr. Charles Gersbach, a pioneer in CRISPR technology at Duke University, a team of biomedical engineers has ident...

A recently published paper explores a novel gene therapy approach for PWS, delivering a ‘growth hormone’ for brain cells, called brain-derived neurotrophic factor (BDNF). The study, Hypothalamic AAV-BDNF gene therapy improves metabolic function and b...