A mutation-independent approach for muscular dystrophy via upregulation of a modifier gene
出版年份 2019 全文链接
标题
A mutation-independent approach for muscular dystrophy via upregulation of a modifier gene
作者
关键词
-
出版物
NATURE
Volume 572, Issue 7767, Pages 125-130
出版商
Springer Science and Business Media LLC
发表日期
2019-07-25
DOI
10.1038/s41586-019-1430-x
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。- Increased polyamines as protective disease modifiers in congenital muscular dystrophy
- (2018) D U Kemaladewi et al. HUMAN MOLECULAR GENETICS
- Laminin α1 reduces muscular dystrophy in dy 2J mice
- (2018) Kinga I. Gawlik et al. MATRIX BIOLOGY
- Amelioration of Muscle and Nerve Pathology in LAMA2 Muscular Dystrophy by AAV9-Mini-Agrin
- (2018) Chunping Qiao et al. Molecular Therapy-Methods & Clinical Development
- Genetic Modulation of RNA Splicing with a CRISPR-Guided Cytidine Deaminase
- (2018) Juanjuan Yuan et al. MOLECULAR CELL
- Treatment of a metabolic liver disease by in vivo genome base editing in adult mice
- (2018) Lukas Villiger et al. NATURE MEDICINE
- Chimeric protein repair of laminin polymerization ameliorates muscular dystrophy phenotype
- (2017) Karen K. McKee et al. JOURNAL OF CLINICAL INVESTIGATION
- Correction of a splicing defect in a mouse model of congenital muscular dystrophy type 1A using a homology-directed-repair-independent mechanism
- (2017) Dwi U Kemaladewi et al. NATURE MEDICINE
- Linker proteins restore basement membrane and correct LAMA2 -related muscular dystrophy in mice
- (2017) Judith R. Reinhard et al. Science Translational Medicine
- Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy
- (2017) Niclas E. Bengtsson et al. Nature Communications
- Increased Expression of Laminin Subunit Alpha 1 Chain by dCas9-VP160
- (2017) Arnaud Perrin et al. Molecular Therapy-Nucleic Acids
- A Five-Repeat Micro-Dystrophin Gene Ameliorated Dystrophic Phenotype in the Severe DBA/2J-mdx Model of Duchenne Muscular Dystrophy
- (2017) Chady H. Hakim et al. Molecular Therapy-Methods & Clinical Development
- Spell Checking Nature: Versatility of CRISPR/Cas9 for Developing Treatments for Inherited Disorders
- (2016) Daria Wojtal et al. AMERICAN JOURNAL OF HUMAN GENETICS
- In vivo genome editing using Staphylococcus aureus Cas9
- (2015) F. Ann Ran et al. NATURE
- Highly specific epigenome editing by CRISPR-Cas9 repressors for silencing of distal regulatory elements
- (2015) Pratiksha I Thakore et al. NATURE METHODS
- Diagnostic approach to the congenital muscular dystrophies
- (2014) Carsten G. Bönnemann et al. NEUROMUSCULAR DISORDERS
- RNA-guided gene activation by CRISPR-Cas9–based transcription factors
- (2013) Pablo Perez-Pinera et al. NATURE METHODS
- CRISPR RNA–guided activation of endogenous human genes
- (2013) Morgan L Maeder et al. NATURE METHODS
- Peripheral nerve pathology, including aberrant Schwann cell differentiation, is ameliorated by doxycycline in a laminin- 2-deficient mouse model of congenital muscular dystrophy
- (2011) S. Homma et al. HUMAN MOLECULAR GENETICS
- Notch1 signaling plays a role in regulating precursor differentiation during CNS remyelination
- (2009) Yueting Zhang et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Laminin-111 protein therapy prevents muscle disease in the mdx mouse model for Duchenne muscular dystrophy
- (2009) J. E. Rooney et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- A single point mutation in the LN domain of LAMA2 causes muscular dystrophy and peripheral amyelination
- (2008) B. L. Patton et al. JOURNAL OF CELL SCIENCE
Discover Peeref hubs
Discuss science. Find collaborators. Network.
Join a conversationCreate your own webinar
Interested in hosting your own webinar? Check the schedule and propose your idea to the Peeref Content Team.
Create Now