“The authors demonstrate the long-term safety and durability of an adeno-associated viral vector serotype 5 encoding an engineered miRNA (AAV5-miHTT) targeting human mutant huntingtin (mHTT) in the minipig animal model of Huntington’s disease (HD) up to 5 years post-injection,” Jennifer Goldman, MD, MS, expert movement disorder neurologist. “This finding expands on their prior work revealing sustained cerebrospinal fluid (CSF) mHTT protein lowering up to 4 years after a one-time intrastriatal administration of AAV5-miHTT.”
“HD is an inherited neurodegenerative disorder for which we still seek a cure or disease-modifying treatment. As such, HTT-lowering strategies have received growing interest for their disease-modifying potential in HD clinical trials. AAV5 has been a candidate for RNAi-based gene transfer in HD animal models, and adeno-associated viral vectors have been recently studied in early-phase HD human trials, while others have been approved for neurologic diseases such as spinal muscle atrophy. The study illustrates an important sustained duration of effect on mHTT post-injection.
“In addition, the ability to deliver AAV5 intrathecally rather than directly via striatal delivery, coupled with demonstrated effects on mHTT in this abstract, may have promise for future human trials with therapeutic delivery via lumbar puncture infusion rather than neurosurgical stereotactical procedures.
“Whether changes in protein levels as measured in the brain, CSF or other biofluids are associated with suppression of aggregate formations, prevention of neuronal dysfunction, or importantly lead to clinical and behavioral changes and functional improvements remain to be seen. Understanding the effects of reduced mHTT and the targeted intervention on different brain regions will be important considerations in translating to human therapies. This research also adds to the literature regarding large animal models under study for HD such as minipigs, which may offer promise beyond rodent models for drug development. However, further development in these models regarding clinical features and behavioral tasks of HD may be needed.”
View the full-text abstract:
http://www.mdsabstracts.org – Reference #: 865
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