Successful Huntington’s disease intervention reported by Sangamo

Country

United States

A group of international scientists has successfully repressed the transcription of the mutant gene causing Huntington’s disease, enabling animal models with the disease to show clear improvements in a range of important functions. Results of the research were published online on 1 July in the journal Nature Medicine. The studies were carried out by Sangamo Therapeutics Inc and the CHDI Foundation, a non-profit organisation devoted to Huntington’s disease research.