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Deficiency in the GBA1 encoded enzyme glucocerebrosidase (GCase) leads to the accumulation of the GCase glycolipid substrates glucosylceramide and glucosylsphingosine and ultimately results in toxicity and inflammation and negatively affect many clinical aspects of Parkinson\u2019s disease, including disease risk, the severity of presentation, age of onset, and likelihood of progression to dementia. These findings support the view that re-establishing normal levels of GCase enzyme activity may reduce the progression of Parkinson\u2019s disease in patients carrying GBA1 mutations. Studies in mouse models indicate that PR001, a AAV9 vector-based gene therapy designed to deliver a functional GBA1 gene to the brain, suggest that this therapeutic approach may slow or stop disease progression. PR001 is currently being evaluated in clinical trials with Parkinson\u2019s disease patients carrying GBA1 mutations.<\/jats:p>","DOI":"10.3233\/jpd-212739","type":"journal-article","created":{"date-parts":[[2021,6,15]],"date-time":"2021-06-15T14:10:02Z","timestamp":1623766202000},"page":"S183-S188","source":"Crossref","is-referenced-by-count":63,"title":["Gene Therapy for Parkinson\u2019s Disease Associated with GBA1 Mutations"],"prefix":"10.1177","volume":"11","author":[{"given":"Asa","family":"Abeliovich","sequence":"first","affiliation":[{"name":"Prevail Therapeutics, A Wholly-Owned Subsidiary of Eli Lilly and Company, New York, NY, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Franz","family":"Hefti","sequence":"additional","affiliation":[{"name":"Prevail Therapeutics, A Wholly-Owned Subsidiary of Eli Lilly and Company, New York, NY, 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