用于治疗肌肉萎缩症的病毒基因疗法的第一步。 - Suppr

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A First Step in Viral Gene Therapy for Muscular Dystrophy.

作者信息

Hirano Michio

机构信息

Columbia University Medical Center, 630 West 168th Street, P&S 4-423, New York, NY 10032, USA.

出版信息

Curr Neurol Neurosci Rep. 2010 Mar;10(2):71-72. doi: 10.1007/s11910-010-0090-x.

DOI:10.1007/s11910-010-0090-x

PMID:20651902

原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC2861776/

Abstract

摘要

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The evolution of gene therapy in X-linked severe combined immunodeficiency.

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Therapy for neuromuscular disorders.

Curr Opin Genet Dev. 2009 Jun;19(3):290-7. doi: 10.1016/j.gde.2009.03.005. Epub 2009 May 4.

A single intravenous injection of adeno-associated virus serotype-9 leads to whole body skeletal muscle transduction in dogs.

Mol Ther. 2008 Dec;16(12):1944-52. doi: 10.1038/mt.2008.207. Epub 2008 Sep 30.

Limb-girdle muscular dystrophies--from genetics to molecular pathology.

Neuropathol Appl Neurobiol. 2004 Apr;30(2):91-105. doi: 10.1111/j.1365-2990.2004.00555.x.

Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning.

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Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease.

Science. 2000 Apr 28;288(5466):669-72. doi: 10.1126/science.288.5466.669.

Dystrophin: the protein product of the Duchenne muscular dystrophy locus.

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相似文献

A First Step in Viral Gene Therapy for Muscular Dystrophy.

Curr Neurol Neurosci Rep. 2010 Mar;10(2):71-72. doi: 10.1007/s11910-010-0090-x.

Gene therapy for duchenne muscular dystrophy: expectations and challenges.

Arch Neurol. 2007 Sep;64(9):1236-41. doi: 10.1001/archneur.64.9.1236.

The Fukuyama congenital muscular dystrophy story.

Neuromuscul Disord. 2000 Mar;10(3):153-9. doi: 10.1016/s0960-8966(99)00109-1.

Emerging strategies for cell and gene therapy of the muscular dystrophies.

Expert Rev Mol Med. 2009 Jun 25;11:e18. doi: 10.1017/S1462399409001100.

Prospect of gene therapy for cardiomyopathy in hereditary muscular dystrophy.

Expert Opin Orphan Drugs. 2016;4(2):169-183. doi: 10.1517/21678707.2016.1124039. Epub 2015 Dec 17.

Gene therapy for selected neuromuscular and trinucleotide repeat disorders - An insight to subsume South Asia for multicenter clinical trials.

IBRO Neurosci Rep. 2023 Jan 30;14:146-153. doi: 10.1016/j.ibneur.2023.01.009. eCollection 2023 Jun.

[Analysis of motor control and kinesthetic perception in patients with progressive muscular dystrophy].

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Curr Opin Neurol. 2012 Oct;25(5):588-96. doi: 10.1097/WCO.0b013e328357b0be.

Comparison of Serum rAAV Serotype-Specific Antibodies in Patients with Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Inclusion Body Myositis, or GNE Myopathy.

Hum Gene Ther. 2017 Sep;28(9):737-746. doi: 10.1089/hum.2016.141. Epub 2016 Dec 29.

New approaches in the therapy of cardiomyopathy in muscular dystrophy.

Annu Rev Med. 2007;58:75-88. doi: 10.1146/annurev.med.58.011706.144703.

引用本文的文献

Screening of mitochondrial mutations and insertion-deletion polymorphism in gestational diabetes mellitus in the Asian Indian population.

Saudi J Biol Sci. 2015 May;22(3):243-8. doi: 10.1016/j.sjbs.2014.11.001. Epub 2014 Nov 12.

本文引用的文献

Gene therapy for muscular dystrophy reaches human clinical trial.

Ann Neurol. 2009 Sep;66(3):267-70. doi: 10.1002/ana.21803.

The evolution of gene therapy in X-linked severe combined immunodeficiency.

Ann Allergy Asthma Immunol. 2009 May;102(5):357-62; quiz 363-5, 402. doi: 10.1016/S1081-1206(10)60504-2.

Therapy for neuromuscular disorders.

Curr Opin Genet Dev. 2009 Jun;19(3):290-7. doi: 10.1016/j.gde.2009.03.005. Epub 2009 May 4.

A single intravenous injection of adeno-associated virus serotype-9 leads to whole body skeletal muscle transduction in dogs.

Mol Ther. 2008 Dec;16(12):1944-52. doi: 10.1038/mt.2008.207. Epub 2008 Sep 30.

Limb-girdle muscular dystrophies--from genetics to molecular pathology.

Neuropathol Appl Neurobiol. 2004 Apr;30(2):91-105. doi: 10.1111/j.1365-2990.2004.00555.x.

Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning.

Science. 2002 Jun 28;296(5577):2410-3. doi: 10.1126/science.1070104.

Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease.

Science. 2000 Apr 28;288(5466):669-72. doi: 10.1126/science.288.5466.669.

Dystrophin: the protein product of the Duchenne muscular dystrophy locus.

Cell. 1987 Dec 24;51(6):919-28. doi: 10.1016/0092-8674(87)90579-4.