A mutation-independent approach for muscular dystrophy via upregulation of a modifier gene
Published 2019 View Full Article
- Home
- Publications
- Publication Search
- Publication Details
Title
A mutation-independent approach for muscular dystrophy via upregulation of a modifier gene
Authors
Keywords
-
Journal
NATURE
Volume 572, Issue 7767, Pages 125-130
Publisher
Springer Science and Business Media LLC
Online
2019-07-25
DOI
10.1038/s41586-019-1430-x
References
Ask authors/readers for more resources
Related references
Note: Only part of the references are listed.- 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
Find Funding. Review Successful Grants.
Explore over 25,000 new funding opportunities and over 6,000,000 successful grants.
ExploreFind the ideal target journal for your manuscript
Explore over 38,000 international journals covering a vast array of academic fields.
Search