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Intrathecally delivering an adeno-associated virus vector encoding an RfxCas13d variant programmed to target superoxide dismutase 1 (SOD1), a protein whose mutation can cause amyotrophic lateral sclerosis, reduced SOD1 mRNA and protein in the spinal cord by &gt;50% and improved outcomes in a mouse model of the disorder. We further show that intrastriatally delivering an RfxCas13d variant programmed to target huntingtin (HTT), a protein whose mutation is causative for Huntington\u2019s disease, led to a ~50% reduction in HTT protein in the mouse brain. Our results establish RfxCas13d as a versatile platform for knocking down gene expression in the nervous system.<\/jats:p>","DOI":"10.1126\/sciadv.abk2485","type":"journal-article","created":{"date-parts":[[2022,1,19]],"date-time":"2022-01-19T13:59:26Z","timestamp":1642600766000},"source":"Crossref","is-referenced-by-count":97,"title":["Targeted gene silencing in the nervous system with CRISPR-Cas13"],"prefix":"10.1126","volume":"8","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-2207-4468","authenticated-orcid":true,"given":"Jackson E.","family":"Powell","sequence":"first","affiliation":[{"name":"Department of Bioengineering, University of Illinois, Urbana, IL 61801, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Colin K. 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