A phase I trial of adeno-associated virus serotype 1-γ-sarcoglycan gene therapy for limb girdle muscular dystrophy type 2C
出版年份 2012 全文链接
标题
A phase I trial of adeno-associated virus serotype 1-γ-sarcoglycan gene therapy for limb girdle muscular dystrophy type 2C
作者
关键词
-
出版物
BRAIN
Volume 135, Issue 2, Pages 483-492
出版商
Oxford University Press (OUP)
发表日期
2012-01-12
DOI
10.1093/brain/awr342
参考文献
相关参考文献
注意:仅列出部分参考文献,下载原文获取全部文献信息。- Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D
- (2010) Jerry R. Mendell et al. ANNALS OF NEUROLOGY
- Evaluation of Hydrodynamic Limb Vein Injections in Nonhuman Primates
- (2010) Julia O. Hegge et al. HUMAN GENE THERAPY
- Prevalence of Serum IgG and Neutralizing Factors Against Adeno-Associated Virus (AAV) Types 1, 2, 5, 6, 8, and 9 in the Healthy Population: Implications for Gene Therapy Using AAV Vectors
- (2010) Sylvie Boutin et al. HUMAN GENE THERAPY
- Dystrophin Immunity in Duchenne's Muscular Dystrophy
- (2010) Jerry R. Mendell et al. NEW ENGLAND JOURNAL OF MEDICINE
- Limb-girdle muscular dystrophy type 2D gene therapy restores α-sarcoglycan and associated proteins
- (2009) Jerry R. Mendell et al. ANNALS OF NEUROLOGY
- AAV-1-mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells
- (2009) F. Mingozzi et al. BLOOD
- Transient Transfection Methods for Clinical Adeno-Associated Viral Vector Production
- (2009) J. Fraser Wright HUMAN GENE THERAPY
- Calcium Upregulation by Percutaneous Administration of Gene Therapy in Cardiac Disease (CUPID Trial), a First-in-Human Phase 1/2 Clinical Trial
- (2009) Brian E. Jaski et al. JOURNAL OF CARDIAC FAILURE
- Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial
- (2009) Albert M Maguire et al. LANCET
- Lack of Immunotoxicity After Regional Intravenous (RI) Delivery of rAAV to Nonhuman Primate Skeletal Muscle
- (2009) Alice Toromanoff et al. MOLECULAR THERAPY
- Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy
- (2009) M. L. Brantly et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Intramuscular Administration of AAV1-Lipoprotein Lipase S447X Lowers Triglycerides in Lipoprotein Lipase–Deficient Patients
- (2008) Erik S. Stroes et al. ARTERIOSCLEROSIS THROMBOSIS AND VASCULAR BIOLOGY
- Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis
- (2008) Albert M. Maguire et al. NEW ENGLAND JOURNAL OF MEDICINE
- Effect of Gene Therapy on Visual Function in Leber's Congenital Amaurosis
- (2008) James W.B. Bainbridge et al. NEW ENGLAND JOURNAL OF MEDICINE
- Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics
- (2008) A. V. Cideciyan et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
Add your recorded webinar
Do you already have a recorded webinar? Grow your audience and get more views by easily listing your recording on Peeref.
Upload NowAsk a Question. Answer a Question.
Quickly pose questions to the entire community. Debate answers and get clarity on the most important issues facing researchers.
Get Started