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2023 | 2 |
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Dwarf Open Reading Frame (DWORF) Gene Therapy Ameliorated Duchenne Muscular Dystrophy Cardiomyopathy in Aged mdx Mice.
J Am Heart Assoc. 2023 Feb 7;12(3):e027480. doi: 10.1161/JAHA.122.027480. Epub 2023 Jan 25.
J Am Heart Assoc. 2023.
PMID: 36695318
Free PMC article.
The gRNA Vector Level Determines the Outcome of Systemic AAV CRISPR Therapy for Duchenne Muscular Dystrophy.
Wasala NB, Million ED, Watkins TB, Wasala LP, Han J, Yue Y, Lu B, Chen SJ, Hakim CH, Duan D.
Wasala NB, et al. Among authors: watkins tb.
Hum Gene Ther. 2022 May;33(9-10):518-528. doi: 10.1089/hum.2021.130. Epub 2022 May 4.
Hum Gene Ther. 2022.
PMID: 35350865
Free PMC article.
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The Implication of Hinge 1 and Hinge 4 in Micro-Dystrophin Gene Therapy for Duchenne Muscular Dystrophy.
Wasala LP, Watkins TB, Wasala NB, Burke MJ, Yue Y, Lai Y, Yao G, Duan D.
Wasala LP, et al. Among authors: watkins tb.
Hum Gene Ther. 2023 May;34(9-10):459-470. doi: 10.1089/hum.2022.180. Epub 2023 Apr 6.
Hum Gene Ther. 2023.
PMID: 36310439
Free PMC article.
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