Gene Therapy for Huntington’s Disease in Development – Rare Disease Report
Huntington’s Disease News |
Gene Therapy for Huntington's Disease in Development
Rare Disease Report In Huntington's disease animal models, the gene therapy called AMT-130, an AAV5 vector carrying a DNA cassette encoding artificial micro-RNA, is effective. In April, data was presented at the 12th Annual CHDI HD Therapeutics Conference in Malta showing … UniQure Plans Tests That Could Lead to Clinical Trial of AMT-130 Gene Therapy for Huntington's |
