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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.
Comparison of different promoters to improve AAV vector-mediated gene therapy for neuronopathic Gaucher disease.
Massaro G, Geard AF, Nelvagal HR, Gore K, Clemo NK, Waddington SN, Rahim AA.
Massaro G, et al. Among authors: geard af.
Hum Mol Genet. 2024 May 16:ddae081. doi: 10.1093/hmg/ddae081. Online ahead of print.
Hum Mol Genet. 2024.
PMID: 38757200
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