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Muscle Gene Therapy最新文献

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What We Have Learned from 10 Years of DMD Exon-Skipping Trials 我们从10年的DMD外显子跳跃试验中学到的东西
Muscle Gene Therapy Pub Date : 1900-01-01 DOI: 10.1007/978-3-030-03095-7_43
S. Pasteuning-Vuhman, A. Aartsma-Rus
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引用次数: 0
Pluripotent Stem Cells for Gene Therapy of Hereditary Muscle Disorders 多能干细胞用于遗传性肌肉疾病的基因治疗
Muscle Gene Therapy Pub Date : 1900-01-01 DOI: 10.1007/978-3-030-03095-7_5
T. VandenDriessche, Y. C. Chai, D. Boon, M. Chuah
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引用次数: 0
An Overview of Muscle Biology and Physiology for Muscle Gene Therapy 肌肉基因治疗的肌肉生物学和生理学综述
Muscle Gene Therapy Pub Date : 1900-01-01 DOI: 10.1007/978-3-030-03095-7_1
P. Janssen, Jonathan P. Davis
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引用次数: 0
Design of Muscle Gene Therapy Expression Cassette 肌肉基因治疗表达盒的设计
Muscle Gene Therapy Pub Date : 1900-01-01 DOI: 10.1007/978-3-030-03095-7_8
Yi Lai, D. Duan
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引用次数: 2
Gene Therapy for X-Linked Myotubular Myopathy x连锁肌小管肌病的基因治疗
Muscle Gene Therapy Pub Date : 1900-01-01 DOI: 10.1007/978-3-030-03095-7_32
J. Dupont, M. Lawlor, M. Childers
{"title":"Gene Therapy for X-Linked Myotubular Myopathy","authors":"J. Dupont, M. Lawlor, M. Childers","doi":"10.1007/978-3-030-03095-7_32","DOIUrl":"https://doi.org/10.1007/978-3-030-03095-7_32","url":null,"abstract":"","PeriodicalId":127683,"journal":{"name":"Muscle Gene Therapy","volume":"554 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"1900-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"117063379","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 2
Patient and Family Perspective on Muscle Gene Therapy 肌肉基因治疗的患者和家庭观点
Muscle Gene Therapy Pub Date : 1900-01-01 DOI: 10.1007/978-3-030-03095-7_37
P. Furlong
{"title":"Patient and Family Perspective on Muscle Gene Therapy","authors":"P. Furlong","doi":"10.1007/978-3-030-03095-7_37","DOIUrl":"https://doi.org/10.1007/978-3-030-03095-7_37","url":null,"abstract":"","PeriodicalId":127683,"journal":{"name":"Muscle Gene Therapy","volume":"3 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"1900-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"121108356","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Path to Clinical Trials: Trial Design, Development of the Clinical Product, and Safety Concerns in the Implementation of Clinical Trials 临床试验之路:试验设计、临床产品的开发和临床试验实施中的安全问题
Muscle Gene Therapy Pub Date : 1900-01-01 DOI: 10.1007/978-3-030-03095-7_39
J. Mendell, L. Rodino-Klapac, C. Shilling
{"title":"Path to Clinical Trials: Trial Design, Development of the Clinical Product, and Safety Concerns in the Implementation of Clinical Trials","authors":"J. Mendell, L. Rodino-Klapac, C. Shilling","doi":"10.1007/978-3-030-03095-7_39","DOIUrl":"https://doi.org/10.1007/978-3-030-03095-7_39","url":null,"abstract":"","PeriodicalId":127683,"journal":{"name":"Muscle Gene Therapy","volume":"21 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"1900-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"132737874","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 1
Genome Editing for Muscle Gene Therapy 肌肉基因治疗的基因组编辑
Muscle Gene Therapy Pub Date : 1900-01-01 DOI: 10.1007/978-3-030-03095-7_16
Alan O'Brien, R. Cohn
{"title":"Genome Editing for Muscle Gene Therapy","authors":"Alan O'Brien, R. Cohn","doi":"10.1007/978-3-030-03095-7_16","DOIUrl":"https://doi.org/10.1007/978-3-030-03095-7_16","url":null,"abstract":"","PeriodicalId":127683,"journal":{"name":"Muscle Gene Therapy","volume":"6 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"1900-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"134334371","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 2
AAV-Mediated Exon Skipping for Duchenne Muscular Dystrophy aav介导的杜氏肌营养不良外显子跳变
Muscle Gene Therapy Pub Date : 1900-01-01 DOI: 10.1007/978-3-030-03095-7_20
R. Benchaouir, A. Goyenvalle
{"title":"AAV-Mediated Exon Skipping for Duchenne Muscular Dystrophy","authors":"R. Benchaouir, A. Goyenvalle","doi":"10.1007/978-3-030-03095-7_20","DOIUrl":"https://doi.org/10.1007/978-3-030-03095-7_20","url":null,"abstract":"","PeriodicalId":127683,"journal":{"name":"Muscle Gene Therapy","volume":"17 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"1900-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"132284157","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 1
Histological and Biochemical Evaluation of Muscle Gene Therapy 肌肉基因治疗的组织学和生化评价
Muscle Gene Therapy Pub Date : 1900-01-01 DOI: 10.1007/978-3-030-03095-7_12
M. Lawlor, Joel S. Schneider, M. Childers, K. Brown
{"title":"Histological and Biochemical Evaluation of Muscle Gene Therapy","authors":"M. Lawlor, Joel S. Schneider, M. Childers, K. Brown","doi":"10.1007/978-3-030-03095-7_12","DOIUrl":"https://doi.org/10.1007/978-3-030-03095-7_12","url":null,"abstract":"","PeriodicalId":127683,"journal":{"name":"Muscle Gene Therapy","volume":"77 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"1900-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"133468852","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
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