Research ArticleHUNTINGTON DISEASE

Widespread and sustained target engagement in Huntington’s disease minipigs upon intrastriatal microRNA-based gene therapy

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Science Translational Medicine  07 Apr 2021:
Vol. 13, Issue 588, eabb8920
DOI: 10.1126/scitranslmed.abb8920

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Targeting HTT in pigs

Huntington’s disease (HD) is a genetic neurodegenerative disorder caused by mutated huntingtin (HTT) gene. Reducing the expression of the aberrant HTT has been shown to be effective in preclinical models. Now, Vallès et al. evaluated the effects of an adeno-associated viral vector (AAV)–mediated strategy delivering microRNA (miRNA) targeting human mutant HTT (mHTT) in a pig model of HD that closely resembles the human condition. Intracerebral delivery of the miRNA into the striatum resulted in widespread distribution and reduced mHTT for up to a year after injection. The results suggest that the approach could be effective in patients with HD.

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