A first-in-human phase I/IIa gene transfer clinical trial for Duchenne muscular dystrophy using rAAVrh74.MCK.GALGT2
出版年份 2022 全文链接
标题
A first-in-human phase I/IIa gene transfer clinical trial for Duchenne muscular dystrophy using rAAVrh74.MCK.GALGT2
作者
关键词
-
出版物
Molecular Therapy-Methods & Clinical Development
Volume 27, Issue -, Pages 47-60
出版商
Elsevier BV
发表日期
2022-09-02
DOI
10.1016/j.omtm.2022.08.009
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。- Duchenne muscular dystrophy
- (2021) Dongsheng Duan et al. Nature Reviews Disease Primers
- Short-term treatment of golden retriever muscular dystrophy (GRMD) dogs with rAAVrh74.MHCK7.GALGT2 induces muscle glycosylation and utrophin expression but has no significant effect on muscle strength
- (2021) Paul T. Martin et al. PLoS One
- Automated immunofluorescence analysis for sensitive and precise dystrophin quantification in muscle biopsies
- (2021) Tatyana A. Vetter et al. NEUROPATHOLOGY AND APPLIED NEUROBIOLOGY
- Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy
- (2020) Diane E. Frank et al. NEUROLOGY
- Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping
- (2020) Paula R. Clemens et al. JAMA Neurology
- Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy
- (2020) Jerry R. Mendell et al. JAMA Neurology
- rAAVrh74.MCK.GALGT2 Protects Against Loss of Hemodynamic Function in the Aging mdx Mouse Heart
- (2019) Rui Xu et al. MOLECULAR THERAPY
- Gene Delivery for Limb-Girdle Muscular Dystrophy Type 2D (LGMD2D)
- (2019) Jerry R. Mendell et al. HUMAN GENE THERAPY
- rAAVrh74.MCK.GALGT2 Demonstrates Safety and Widespread Muscle Glycosylation after Intravenous Delivery in C57BL/6J Mice
- (2019) Deborah A. Zygmunt et al. Molecular Therapy-Methods & Clinical Development
- Systemic AAV micro-dystrophin gene therapy for Duchenne muscular dystrophy
- (2018) Dongsheng Duan MOLECULAR THERAPY
- An Isolated Limb Infusion Method Allows for Broad Distribution of rAAVrh74.MCK. GALGT2 to Leg Skeletal Muscles in the Rhesus Macaque
- (2018) Rui Xu et al. Molecular Therapy-Methods & Clinical Development
- B4GALNT2 (GALGT2) Gene Therapy Reduces Skeletal Muscle Pathology in the FKRP P448L Mouse Model of Limb Girdle Muscular Dystrophy 2I
- (2016) Paul J. Thomas et al. AMERICAN JOURNAL OF PATHOLOGY
- Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy
- (2016) Jerry R. Mendell et al. ANNALS OF NEUROLOGY
- Deletion of Galgt2 (B4Galnt2) Reduces Muscle Growth in Response to Acute Injury and Increases Muscle Inflammation and Pathology in Dystrophin-Deficient Mice
- (2015) Rui Xu et al. AMERICAN JOURNAL OF PATHOLOGY
- Vascular Delivery of rAAVrh74.MCK.GALGT2 to the Gastrocnemius Muscle of the Rhesus Macaque Stimulates the Expression of Dystrophin and Laminin α2 Surrogates
- (2013) Louis G. Chicoine et al. MOLECULAR THERAPY
- Plasmapheresis Eliminates the Negative Impact of AAV Antibodies on Microdystrophin Gene Expression Following Vascular Delivery
- (2013) LG Chicoine et al. MOLECULAR THERAPY
- Evidence-based path to newborn screening for duchenne muscular dystrophy
- (2012) Jerry R. Mendell et al. ANNALS OF NEUROLOGY
- Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D
- (2010) Jerry R. Mendell et al. ANNALS OF NEUROLOGY
- Change in Natural History of Duchenne Muscular Dystrophy With Long-term Corticosteroid Treatment: Implications for Management
- (2010) Richard T. Moxley et al. JOURNAL OF CHILD NEUROLOGY
- Serum Transaminase Levels in Boys With Duchenne and Becker Muscular Dystrophy
- (2010) H. J. McMillan et al. PEDIATRICS
- Overexpression of Galgt2 Reduces Dystrophic Pathology in the Skeletal Muscles of Alpha Sarcoglycan-Deficient Mice
- (2009) Rui Xu et al. AMERICAN JOURNAL OF PATHOLOGY
- Overexpression of Galgt2 in skeletal muscle prevents injury resulting from eccentric contractions in both mdx and wild-type mice
- (2008) Paul T. Martin et al. AMERICAN JOURNAL OF PHYSIOLOGY-CELL PHYSIOLOGY
Discover Peeref hubs
Discuss science. Find collaborators. Network.
Join a conversationFind the ideal target journal for your manuscript
Explore over 38,000 international journals covering a vast array of academic fields.
Search