Gene Therapy Development and Validation for Huntington's Disease Fibro TG-HD.

A clinical trial, NCT06444217, has been launched to develop and validate gene therapy for Huntington's disease. Huntington's disease is a rare and fatal neurodegenerative disorder caused by an expansion of CAG triplets in the Huntingtin gene. The trial aims to use trans-splicing gene therapy to correct mutated gene transcripts and evaluate its therapeutic effects in vitro using fibroblast cell lines derived from skin biopsies of Huntington's disease patients. While there are currently no approved neuroprotective or curative treatments for Huntington's disease, this alternative approach shows promise in selectively replacing mutated sequences with the wild-type gene. [Extracted from the article]