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Image: The field's first peer-reviewed journal, providing comprehensive access to key pillars of human gene therapy: research, methods and clinical applications
Gene therapy targeting the messenger RNA (mRNA) of the huntingtin mutant gene (HTT) can provide long-term therapeutic effects after a single dose
"The challenges of treating autosomal dominant brain disorders are enormous, and this pioneering effort by Voyager Therapeutics is a key step forward for the most tragic type of Huntington's disease," said Terence R.
Dr.
The researchers selected an optimized pri-amiRNA "for AAV gene therapy, showing efficient and precise pri-amiRNA processing, as well as potent HTT-reducing pharmacological activity and general tolerability of in vivo therapy for Huntington's disease
Human Gene Therapy, the official journal of the European Society for Gene and Cell Therapy and eight other international gene therapy societies, is the first peer-reviewed journal in the field, providing insights into the key pillars of gene therapy in humans: research, methods and Comprehensive access to clinical applications
Original title:
Efficient and Precise Processing of the Optimized Primary Artificial MicroRNA in a Huntingtin-Lowering Adeno-Associated Viral Gene Therapy In Vitro and in Mice and Nonhuman Primates
