Selective Targeting of mHTT Presents Novel Therapeutic Approach to Huntington's Disease


Huntington’s disease (HD) is a fatal neurodegenerative disorder that affects around 30,000 people in the US, with another 200,000 at risk of developing the condition. Despite extensive research into HD, there are currently no approved disease-modifying therapies available. Researchers from CHDI Foundation and Sangamo Therapeutics believe the ideal therapeutic will be one that completely eliminates the expression of mHTT without interfering with normal HTT function. In an effort to do just that, they have engineered zinc finger protein transcription factors (ZFP-TFs) to target the pathogenic CAG repeat and selectively lower mHTT as a therapeutic strategy.