Effects of systemic multiexon skipping with peptide-conjugated morpholinos in the heart of a dog model of Duchenne muscular dystrophy
出版年份 2017 全文链接
标题
Effects of systemic multiexon skipping with peptide-conjugated morpholinos in the heart of a dog model of Duchenne muscular dystrophy
作者
关键词
-
出版物
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
Volume 114, Issue 16, Pages 4213-4218
出版商
Proceedings of the National Academy of Sciences
发表日期
2017-04-04
DOI
10.1073/pnas.1613203114
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。- Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy
- (2016) Jerry R. Mendell et al. ANNALS OF NEUROLOGY
- Deletion of exons 3−9 encompassing a mutational hot spot in the DMD gene presents an asymptomatic phenotype, indicating a target region for multiexon skipping therapy
- (2016) Akinori Nakamura et al. JOURNAL OF HUMAN GENETICS
- Safe and bodywide muscle transduction in young adult Duchenne muscular dystrophy dogs with adeno-associated virus
- (2015) Yongping Yue et al. HUMAN MOLECULAR GENETICS
- Intra-Amniotic rAAV-Mediated Microdystrophin Gene Transfer Improves Canine X-Linked Muscular Dystrophy and May Induce Immune Tolerance
- (2015) Hiromi Hayashita-Kinoh et al. MOLECULAR THERAPY
- Development and Application of an Ultrasensitive Hybridization-Based ELISA Method for the Determination of Peptide-Conjugated Phosphorodiamidate Morpholino Oligonucleotides
- (2015) Umar Burki et al. Nucleic Acid Therapeutics
- Long-Term Efficacy of Systemic Multiexon Skipping Targeting Dystrophin Exons 45–55 With a Cocktail of Vivo-Morpholinos in Mdx52 Mice
- (2015) Yusuke Echigoya et al. Molecular Therapy-Nucleic Acids
- In Silico Screening Based on Predictive Algorithms as a Design Tool for Exon Skipping Oligonucleotides in Duchenne Muscular Dystrophy
- (2015) Yusuke Echigoya et al. PLoS One
- Duchenne and Becker Muscular Dystrophies
- (2014) Kevin M. Flanigan NEUROLOGIC CLINICS
- Skipping Multiple Exons of Dystrophin Transcripts Using Cocktail Antisense Oligonucleotides
- (2014) Yusuke Echigoya et al. Nucleic Acid Therapeutics
- Long-Term Rescue of Dystrophin Expression and Improvement in Muscle Pathology and Function in Dystrophic mdx Mice by Peptide-Conjugated Morpholino
- (2012) Bo Wu et al. AMERICAN JOURNAL OF PATHOLOGY
- Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery
- (2012) Y. Aoki et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Pip6-PMO, A New Generation of Peptide-oligonucleotide Conjugates With Improved Cardiac Exon Skipping Activity for DMD Treatment
- (2012) Corinne Betts et al. Molecular Therapy-Nucleic Acids
- Cardiomyopathy of duchenne muscular dystrophy: Current understanding and future directions
- (2011) Christopher F. Spurney MUSCLE & NERVE
- Morpholinos and their peptide conjugates: Therapeutic promise and challenge for Duchenne muscular dystrophy
- (2010) Hong M. Moulton et al. BIOCHIMICA ET BIOPHYSICA ACTA-BIOMEMBRANES
- Diaphragm rescue alone prevents heart dysfunction in dystrophic mice
- (2010) Alastair Crisp et al. HUMAN MOLECULAR GENETICS
- In-frame Dystrophin Following Exon 51-Skipping Improves Muscle Pathology and Function in the Exon 52–Deficient mdx Mouse
- (2010) Yoshitsugu Aoki et al. MOLECULAR THERAPY
- Widespread Muscle Expression of an AAV9 Human Mini-dystrophin Vector After Intravenous Injection in Neonatal Dystrophin-deficient Dogs
- (2010) Joe N Kornegay et al. MOLECULAR THERAPY
- One-year Treatment of Morpholino Antisense Oligomer Improves Skeletal and Cardiac Muscle Functions in Dystrophic mdx Mice
- (2010) Bo Wu et al. MOLECULAR THERAPY
- Efficacy of systemic morpholino exon-skipping in duchenne dystrophy dogs
- (2009) Toshifumi Yokota et al. ANNALS OF NEUROLOGY
- Long-term improvement in mdx cardiomyopathy after therapy with peptide-conjugated morpholino oligomers†
- (2009) Natee Jearawiriyapaisarn et al. CARDIOVASCULAR RESEARCH
- Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations
- (2009) Annemieke Aartsma-Rus et al. HUMAN MUTATION
- Mammalian animal models for Duchenne muscular dystrophy
- (2009) Raffaella Willmann et al. NEUROMUSCULAR DISORDERS
- Selective Vacuolar Degeneration in Dystrophin-Deficient Canine Purkinje Fibers Despite Preservation of Dystrophin-Associated Proteins With Overexpression of Dp71
- (2008) Nobuyuki Urasawa et al. CIRCULATION
- Sustained Dystrophin Expression Induced by Peptide-conjugated Morpholino Oligomers in the Muscles of mdx Mice
- (2008) Natee Jearawiriyapaisarn et al. MOLECULAR THERAPY
- Effective rescue of dystrophin improves cardiac function in dystrophin-deficient mice by a modified morpholino oligomer
- (2008) B. Wu et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
Discover Peeref hubs
Discuss science. Find collaborators. Network.
Join a conversationAsk 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