Full-length dystrophin gene therapy for Duchenne muscular dystrophy.
作者信息
Duan Dongsheng
机构信息
Department of Molecular Microbiology and Immunology and Department of Neurology, School of Medicine, Department of Biomedical Sciences, College of Veterinary Medicine; Department of Chemical and Biomedical Engineering, College of Engineering, University of Missouri, Columbia, MO 65212, USA.
出版信息
Mol Ther. 2024 Sep 4;32(9):2817-2818. doi: 10.1016/j.ymthe.2024.07.026. Epub 2024 Aug 12.
Abstract
摘要
相似文献
1
Full-length dystrophin gene therapy for Duchenne muscular dystrophy.
Mol Ther. 2024 Sep 4;32(9):2817-2818. doi: 10.1016/j.ymthe.2024.07.026. Epub 2024 Aug 12.
2
Full-length dystrophin restoration via targeted exon integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy.
Mol Ther. 2021 Nov 3;29(11):3243-3257. doi: 10.1016/j.ymthe.2021.09.003. Epub 2021 Sep 10.
3
The road toward AAV-mediated gene therapy of Duchenne muscular dystrophy.
Mol Ther. 2025 May 7;33(5):2035-2051. doi: 10.1016/j.ymthe.2025.03.065. Epub 2025 Apr 2.
4
Micro-Dystrophin Gene Therapy Goes Systemic in Duchenne Muscular Dystrophy Patients.
Hum Gene Ther. 2018 Jul;29(7):733-736. doi: 10.1089/hum.2018.012. Epub 2018 Apr 5.
5
Recent developments in Duchenne muscular dystrophy: facts and numbers.
J Cachexia Sarcopenia Muscle. 2017 Oct;8(5):681-685. doi: 10.1002/jcsm.12245.
6
Adeno-Associated Virus (AAV) Mediated Dystrophin Gene Transfer Studies and Exon Skipping Strategies for Duchenne Muscular Dystrophy (DMD).
Curr Gene Ther. 2015;15(4):395-415. doi: 10.2174/1566523215666150710123830.
7
Current protocol of a research phase I clinical trial of full-length dystrophin plasmid DNA in Duchenne/Becker muscular dystrophies. Part I: rationale.
Neuromuscul Disord. 2002 Oct;12 Suppl 1:S49-51. doi: 10.1016/s0960-8966(02)00082-2.
8
Therapeutic Applications of CRISPR/Cas for Duchenne Muscular Dystrophy.
Curr Gene Ther. 2017;17(4):301-308. doi: 10.2174/1566523217666171121165046.
9
Myodys, a full-length dystrophin plasmid vector for Duchenne and Becker muscular dystrophy gene therapy.
Curr Opin Mol Ther. 2008 Feb;10(1):86-94.
10
Autologous Cell Therapy Approach for Duchenne Muscular Dystrophy using PiggyBac Transposons and Mesoangioblasts.
Mol Ther. 2018 Apr 4;26(4):1093-1108. doi: 10.1016/j.ymthe.2018.01.021. Epub 2018 Feb 2.
引用本文的文献
1
siRNA Therapeutics for the Treatment of Hereditary Diseases and Other Conditions: A Review.
Int J Mol Sci. 2025 Sep 5;26(17):8651. doi: 10.3390/ijms26178651.
本文引用的文献
1
Systemic delivery of full-length dystrophin in Duchenne muscular dystrophy mice.
Nat Commun. 2024 Jul 21;15(1):6141. doi: 10.1038/s41467-024-50569-6.
2
Split intein-mediated protein trans-splicing to express large dystrophins.
Nature. 2024 Aug;632(8023):192-200. doi: 10.1038/s41586-024-07710-8. Epub 2024 Jul 17.
3
Inteins: A Swiss army knife for synthetic biology.
Biotechnol Adv. 2024 Jul-Aug;73:108349. doi: 10.1016/j.biotechadv.2024.108349. Epub 2024 Mar 27.
4
Immune Responses to Muscle-Directed Adeno-Associated Viral Gene Transfer in Clinical Studies.
Hum Gene Ther. 2023 May;34(9-10):365-371. doi: 10.1089/hum.2023.056.
5
Directed evolution of a family of AAV capsid variants enabling potent muscle-directed gene delivery across species.
Cell. 2021 Sep 16;184(19):4919-4938.e22. doi: 10.1016/j.cell.2021.08.028. Epub 2021 Sep 9.
6
Proteomic analysis identifies key differences in the cardiac interactomes of dystrophin and micro-dystrophin.
Hum Mol Genet. 2021 Jun 26;30(14):1321-1336. doi: 10.1093/hmg/ddab133.
7
Duchenne muscular dystrophy.
Nat Rev Dis Primers. 2021 Feb 18;7(1):13. doi: 10.1038/s41572-021-00248-3.
8
Identification of a myotropic AAV by massively parallel in vivo evaluation of barcoded capsid variants.
Nat Commun. 2020 Oct 28;11(1):5432. doi: 10.1038/s41467-020-19230-w.
9
Full-length dystrophin reconstitution with adeno-associated viral vectors.
Hum Gene Ther. 2014 Jun;25(6):552-62. doi: 10.1089/hum.2013.210. Epub 2014 Mar 31.
10
Triple trans-splicing adeno-associated virus vectors capable of transferring the coding sequence for full-length dystrophin protein into dystrophic mice.
Hum Gene Ther. 2014 Feb;25(2):98-108. doi: 10.1089/hum.2013.164. Epub 2013 Dec 19.
Zotero 插件