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Journal ArticleOPEN ACCESS

PR001 gene therapy improved phenotypes in models of Parkinson’s disease with GBA1 mutation

  • Sheehan P
  • Heckman L
  • Fenn T
  • et al.
Alzheimer's & Dementia (2020) 16(S2)
DOI: 10.1002/alz.043614
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Abstract

Background: Mutations in GBA1, which result in deficiency of lysosomal enzyme glu

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Sheehan, P., Heckman, L. D., Fenn, T., Wong, L. C., Nelson, S., Garimalla, S., … Abeliovich, A. (2020). PR001 gene therapy improved phenotypes in models of Parkinson’s disease with GBA1 mutation. Alzheimer’s & Dementia, 16(S2). https://doi.org/10.1002/alz.043614

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