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Comparison of different promoters to improve AAV vector-mediated gene therapy for neuronopathic Gaucher disease.
Hum Mol Genet. 2024 May 16:ddae081. doi: 10.1093/hmg/ddae081. Online ahead of print.
Hum Mol Genet. 2024.
PMID: 38757200
Gene Therapy for Mitochondrial Diseases: Current Status and Future Perspective.
Di Donfrancesco A, Massaro G, Di Meo I, Tiranti V, Bottani E, Brunetti D.
Di Donfrancesco A, et al.
Pharmaceutics. 2022 Jun 17;14(6):1287. doi: 10.3390/pharmaceutics14061287.
Pharmaceutics. 2022.
PMID: 35745859
Free PMC article.
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