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Page 1
Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development.
Biomolecules. 2021 Apr 20;11(4):611. doi: 10.3390/biom11040611.
Biomolecules. 2021.
PMID: 33924076
Free PMC article.
Review.
A Novel Small NPC1 Promoter Enhances AAV-Mediated Gene Therapy in Mouse Models of Niemann-Pick Type C1 Disease.
Hughes MP, Nelvagal HR, Coombe-Tennant O, Smith D, Smith C, Massaro G, Poupon-Bejuit L, Platt FM, Rahim AA.
Hughes MP, et al. Among authors: coombe tennant o.
Cells. 2023 Jun 13;12(12):1619. doi: 10.3390/cells12121619.
Cells. 2023.
PMID: 37371089
Free PMC article.
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