Deletion of Exon 51 in a Humanized Duchenne Muscular Dystrophy Mouse Model Abolishes Ventricular Arrhythmia Predisposition.
作者信息
Perelli Robin M, Sitton Madeleine J, Bohning Joel D, Pickar-Oliver Adrian, McCullough K Tyler, Moya-Mendez Mary E, Zheng Scott, Daniels Heather, Devlin Garth, Asokan Aravind, Gersbach Charles A, Landstrom Andrew P
机构信息
Department of Cell Biology (R.M.P., C.A.G., A.P.L.), Duke University, Durham, NC.
Department of Biomedical Engineering (M.J.S., J.D.B., A.P.-O., K.T.M.C., H.D., A.A., C.A.G.), Duke University, Durham, NC.
出版信息
Circ Genom Precis Med. 2025 Jun;18(3):e004867. doi: 10.1161/CIRCGEN.124.004867. Epub 2025 Apr 10.
Abstract
摘要
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本文引用的文献
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One in five patients with Duchenne muscular dystrophy dies from other causes than cardiac or respiratory failure.五分之一的杜氏肌营养不良症患者并非死于心脏或呼吸衰竭等原因。
Eur J Epidemiol. 2022 Feb;37(2):147-156. doi: 10.1007/s10654-021-00819-4. Epub 2021 Nov 21.
2
Full-length dystrophin restoration via targeted exon integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy.通过靶向 exon 整合的 AAV-CRISPR 在杜氏肌营养不良症的人源化小鼠模型中实现全长 dystrophin 修复。
Mol Ther. 2021 Nov 3;29(11):3243-3257. doi: 10.1016/j.ymthe.2021.09.003. Epub 2021 Sep 10.
3
Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy.AAV-CRISPR 基因组编辑治疗杜氏肌营养不良症的长期评估。
Nat Med. 2019 Mar;25(3):427-432. doi: 10.1038/s41591-019-0344-3. Epub 2019 Feb 18.
4
Inhibition of CaMKII phosphorylation of RyR2 prevents inducible ventricular arrhythmias in mice with Duchenne muscular dystrophy.抑制 RyR2 的 CaMKII 磷酸化可预防杜氏肌营养不良症小鼠的诱发性室性心律失常。
Heart Rhythm. 2013 Apr;10(4):592-9. doi: 10.1016/j.hrthm.2012.12.016. Epub 2012 Dec 12.
5
Generation and characterization of transgenic mice with the full-length human DMD gene.携带全长人DMD基因的转基因小鼠的产生与特性分析
J Biol Chem. 2008 Feb 29;283(9):5899-907. doi: 10.1074/jbc.M709410200. Epub 2007 Dec 13.
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