Dystrophinopathic subjects with a specific mega-deletion of exons 45-55 in the DMD gene, as a template for CRISPR/Cas9 therapy in Duchenne muscular dystrophy

Poyatos-Garcia J.; Soblechero-Martin P.; Albiasu-Arteta E.; Arechavala-Gomeza V.; Vazquez-Manrique R.; Muelas N.; Marti P.; Gomis C.; Vilchez J.

首页> 外文期刊>Neuromuscular disorders: NMD >Dystrophinopathic subjects with a specific mega-deletion of exons 45-55 in the DMD gene, as a template for CRISPR/Cas9 therapy in Duchenne muscular dystrophy

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Dystrophinopathic subjects with a specific mega-deletion of exons 45-55 in the DMD gene, as a template for CRISPR/Cas9 therapy in Duchenne muscular dystrophy

机译：Dystophinathic受试者在DMD基因中具有特异性Mega-缺失的外显子45-55，作为Duchenne肌营养不良症的CRISPR / CAS9治疗的模板

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《Neuromuscular disorders: NMD》 |2019年第1期|共2页
作者
Poyatos-Garcia J.; Soblechero-Martin P.; Albiasu-Arteta E.; Arechavala-Gomeza V.; Vazquez-Manrique R.; Muelas N.; Marti P.; Gomis C.; Vilchez J.;
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Hosp La Fe Med Res Inst Valencia Spain;

IIS Biocruces Bizkaia Baracaldo Spain;

IIS Biocruces Bizkaia Baracaldo Spain;

IIS Biocruces Bizkaia Baracaldo Spain;

Hosp La Fe Med Res Inst Valencia Spain;

Hosp La Fe Med Res Inst Valencia Spain;

Hosp La Fe Med Res Inst Valencia Spain;

Hosp La Fe Med Res Inst Valencia Spain;

Hosp La Fe Med Res Inst Valencia Spain;

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正文语种 eng
中图分类神经病学;
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1. Dystrophinopathic subjects with a specific mega-deletion of exons 45-55 in the DMD gene, as a template for CRISPR/Cas9 therapy in Duchenne muscular dystrophy [J] . Poyatos-Garcia J., Soblechero-Martin P., Albiasu-Arteta E., Neuromuscular disorders: NMD . 2019,第Suppla1期

机译：Dystophinathic受试者在DMD基因中具有特异性Mega-缺失的外显子45-55，作为Duchenne肌营养不良症的CRISPR / CAS9治疗的模板
2. Follow-up of three patients with a large in-frame deletion of exons 45-55 in the Duchenne muscular dystrophy (DMD) gene. [J] . Nakamura A, Yoshida K, Fukushima K, Journal of clinical neuroscience: official journal of the Neurosurgical Society of Australasia . 2008,第7期

机译：对三例杜兴氏肌营养不良症（DMD）基因中外显子45-55大框内缺失的患者的随访。
3. Cardiac lesions in Duchenne muscular dystrophy model rats with out-of-frame Dmd gene mutation mediated by CRISPR/Cas9 system [J] . Mao Miyamoto, Ryota Tochinai, Shin-ich Sekizawa, Journal of Toxicologic Pathology . 2020,第4期

机译：Duchenne肌营养不良模型大鼠心脏病变与由CRISPR / CAS9系统介导的帧外DMD基因突变的大鼠
4. Dystrophin quantification through high sensitivity mass spectrometry: A novel approach to measure efficacy of exon skipping therapies in Duchenne Muscular Dystrophy [C] . Ken Fantom, Andy West, James Kew American Society for Mass Spectrometry Conference on Mass Spectrometry and Allied Topics . 2012

机译：通过高灵敏度质谱法定量营养不良蛋白定量：一种测量外显子肌营养不良疗效疗效的新方法
5. Improving stem cell-based therapy and developing a novel gene therapy approach for treating Duchenne Muscular Dystrophy (DMD). [D] . Tabebordbar, Mohammadsharif. 2016

机译：改进基于干细胞的疗法，并开发一种新型的基因疗法来治疗杜兴氏肌营养不良症（DMD）。
6. Corrigendum: Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy [O] . Niclas E. Bengtsson, John K. Hall, Guy L. Odom, -1

机译：更正：肌肉特异的CRISPR / Cas9肌营养不良蛋白基因编辑改善了杜氏肌营养不良症小鼠模型的病理生理
7. Cardiac lesions in Duchenne muscular dystrophy model rats with out-of-frame Dmd gene mutation mediated by CRISPR/Cas9 system [O] . Mao Miyamoto, Ryota Tochinai, Shin-ich Sekizawa, 2020

机译：Duchenne肌肉营养不良模型大鼠的心脏病变与框架外框架 DMD 由CRISPR / CAS9系统介导的基因突变
8. Translational Studies of GALGT2 Gene Therapy for Duchenne Muscular Dystrophy. [R] . Martin, P. T. 2014

机译：GaLGT2基因治疗Duchenne肌营养不良症的转化研究。

Dystrophinopathic subjects with a specific mega-deletion of exons 45-55 in the DMD gene, as a template for CRISPR/Cas9 therapy in Duchenne muscular dystrophy

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