Gene Therapy Approach Reported to Block Mutant Huntington’s Gene in Preclinical Studies
Mar 30, 2016 07:00 am | Malika Ammam, PhD
uniQure recently published results from preclinical studies of its gene therapy program, AMT-130, indicating that a one-time administration of AAV5-delivered therapy into the central nervous system can block the mutant HTT gene that causes Huntington’s disease (HD). These findings, in the article “Design, Characterization, and Lead Selection of Therapeutic miRNAs Targeting Huntingtin for Development of Gene Therapy for […]
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