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Yongping Yue

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Articles 84
Citations 3059
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Recent Articles
11.
Pan X, Yue Y, Boftsi M, Wasala L, Tran N, Zhang K, et al.
Gene Ther . 2021 Oct; 29(6):333-345. PMID: 34611321
Inverted terminal repeats (ITRs) are the only wild-type components retained in the genome of adeno-associated virus (AAV) vectors. To determine whether ITR modification is a viable approach for AAV vector...
12.
Wang H, Marrosu E, Brayson D, Wasala N, Johnson E, Scott C, et al.
Hum Mol Genet . 2021 May; 30(14):1321-1336. PMID: 33949649
ΔR4-R23/ΔCT micro-dystrophin (μDys) is a miniaturized version of dystrophin currently evaluated in a Duchenne muscular dystrophy (DMD) gene therapy trial to treat skeletal and cardiac muscle disease. In pre-clinical studies,...
13.
Zhao J, Yue Y, Patel A, Wasala L, Karp J, Zhang K, et al.
Mol Ther Methods Clin Dev . 2020 Sep; 18:856-868. PMID: 32953935
Adeno-associated virus (AAV) is one of the most important gene delivery vehicles for gene therapy. Intramuscular (i.m.) and intravascular (i.v.) injection are commonly used for AAV gene transfer. Unfortunately, the...
14.
Hakim C, Clement N, Wasala L, Yang H, Yue Y, Zhang K, et al.
Mol Ther Methods Clin Dev . 2020 Aug; 18:664-678. PMID: 32775499
Vector production scale-up is a major barrier in systemic adeno-associated virus (AAV) gene therapy. Many scalable manufacturing methods have been developed. However, the potency of the vectors generated by these...
15.
Chiao Y, Zhang H, Sweetwyne M, Whitson J, Ting Y, Basisty N, et al.
Elife . 2020 Jul; 9. PMID: 32648542
Diastolic dysfunction is a prominent feature of cardiac aging in both mice and humans. We show here that 8-week treatment of old mice with the mitochondrial targeted peptide SS-31 (elamipretide)...
16.
Wasala N, Yue Y, Lostal W, Wasala L, Niranjan N, Hajjar R, et al.
Mol Ther . 2020 Jan; 28(3):845-854. PMID: 31981493
Loss of dystrophin leads to Duchenne muscular dystrophy (DMD). A pathogenic feature of DMD is the significant elevation of cytosolic calcium. Supraphysiological calcium triggers protein degradation, membrane damage, and eventually...
17.
Nance M, Shi R, Hakim C, Wasala N, Yue Y, Pan X, et al.
Mol Ther . 2019 Jul; 27(9):1568-1585. PMID: 31327755
CRISPR editing of muscle stem cells (MuSCs) with adeno-associated virus serotype-9 (AAV9) holds promise for sustained gene repair therapy for muscular dystrophies. However, conflicting evidence exists on whether AAV9 transduces...
18.
Zhao J, Yang H, Wasala L, Zhang K, Yue Y, Duan D, et al.
Mol Med . 2019 Jul; 25(1):31. PMID: 31266455
Background: Delocalization of neuronal nitric oxide synthase (nNOS) from the sarcolemma leads to functional muscle ischemia. This contributes to the pathogenesis in cachexia, aging and muscular dystrophy. Mutations in the...
19.
Pan X, Sands S, Yue Y, Zhang K, Levine S, Duan D
Hum Gene Ther . 2019 Jun; 30(9):1039-1051. PMID: 31184217
Krabbe disease is an inherited neurodegenerative disease caused by mutations in the gene. In the infantile form, patients die before 3 years of age. Systemic adeno-associated virus serotype 9 (AAV9)...
20.
Wasala L, Hakim C, Yue Y, Yang N, Duan D
Methods Mol Biol . 2019 Feb; 1937:281-294. PMID: 30706404
Many diseases affect multiple tissues and/or organ systems, or affect tissues that are broadly distributed. For these diseases, an effective gene therapy will require systemic delivery of the therapeutic vector...