标题
Gene therapies in canine models for Duchenne muscular dystrophy
作者
关键词
-
出版物
HUMAN GENETICS
Volume -, Issue -, Pages -
出版商
Springer Nature
发表日期
2019-02-07
DOI
10.1007/s00439-019-01976-z
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。- AAVrh10 Gene Therapy Ameliorates Central and Peripheral Nervous System Disease in Canine Globoid Cell Leukodystrophy (Krabbe Disease)
- (2018) Allison M. Bradbury et al. HUMAN GENE THERAPY
- Glucose Metabolism as a Pre-clinical Biomarker for the Golden Retriever Model of Duchenne Muscular Dystrophy
- (2018) Sarah Morar Schneider et al. MOLECULAR IMAGING AND BIOLOGY
- Systemic administration of the antisense oligonucleotide NS-065/NCNP-01 for skipping of exon 53 in patients with Duchenne muscular dystrophy
- (2018) Hirofumi Komaki et al. Science Translational Medicine
- Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy
- (2018) Leonela Amoasii et al. SCIENCE
- Extensive and Prolonged Restoration of Dystrophin Expression with Vivo-Morpholino-Mediated Multiple Exon Skipping in Dystrophic Dogs
- (2018) Toshifumi Yokota et al. Nucleic Acid Therapeutics
- Considering aspects of the 3Rs principles within experimental animal biology
- (2017) Lynne U. Sneddon et al. JOURNAL OF EXPERIMENTAL BIOLOGY
- Effects of systemic multiexon skipping with peptide-conjugated morpholinos in the heart of a dog model of Duchenne muscular dystrophy
- (2017) Yusuke Echigoya et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Corrigendum: 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
- Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy
- (2017) Caroline Le Guiner et al. Nature Communications
- The golden retriever model of Duchenne muscular dystrophy
- (2017) Joe N. Kornegay Skeletal Muscle
- CRISPR-Cpf1 correction of muscular dystrophy mutations in human cardiomyocytes and mice
- (2017) Yu Zhang et al. Science Advances
- Canine models of inherited bleeding disorders in the development of coagulation assays, novel protein replacement and gene therapies
- (2016) T. C. Nichols et al. JOURNAL OF THROMBOSIS AND HAEMOSTASIS
- In vivo genome editing via CRISPR/Cas9 mediated homology-independent targeted integration
- (2016) Keiichiro Suzuki et al. NATURE
- Use of the six-minute walk test to characterize golden retriever muscular dystrophy
- (2016) Austin R. Acosta et al. NEUROMUSCULAR DISORDERS
- Successful Phenotype Improvement following Gene Therapy for Severe Hemophilia A in Privately Owned Dogs
- (2016) Mary Beth Callan et al. PLoS One
- Safe and bodywide muscle transduction in young adult Duchenne muscular dystrophy dogs with adeno-associated virus
- (2015) Yongping Yue et al. HUMAN MOLECULAR GENETICS
- The TREAT-NMD DMD Global Database: Analysis of More than 7,000 Duchenne Muscular Dystrophy Mutations
- (2015) Catherine L. Bladen et al. HUMAN MUTATION
- Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy
- (2015) C. Long et al. SCIENCE
- In vivo gene editing in dystrophic mouse muscle and muscle stem cells
- (2015) M. Tabebordbar et al. SCIENCE
- Gene therapy for Duchenne muscular dystrophy
- (2015) Julian Ramos et al. Expert Opinion on Orphan Drugs
- 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
- Pharmacologic Management of Duchenne Muscular Dystrophy: Target Identification and Preclinical Trials
- (2014) J. N. Kornegay et al. ILAR JOURNAL
- Skipping Multiple Exons of Dystrophin Transcripts Using Cocktail Antisense Oligonucleotides
- (2014) Yusuke Echigoya et al. Nucleic Acid Therapeutics
- History of gene therapy
- (2013) Thomas Wirth et al. GENE
- Gene Therapy in Large Animal Models of Muscular Dystrophy
- (2013) Z. Wang et al. ILAR JOURNAL
- Reading Frame Correction by Targeted Genome Editing Restores Dystrophin Expression in Cells From Duchenne Muscular Dystrophy Patients
- (2013) David G Ousterout et al. MOLECULAR THERAPY
- Characteristics of magnetic resonance imaging biomarkers in a natural history study of golden retriever muscular dystrophy
- (2013) Zheng Fan et al. NEUROMUSCULAR DISORDERS
- Canine models of Duchenne muscular dystrophy and their use in therapeutic strategies
- (2012) Joe N. Kornegay et al. MAMMALIAN GENOME
- A call for transparent reporting to optimize the predictive value of preclinical research
- (2012) Story C. Landis et al. NATURE
- 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
- Gene therapy rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa
- (2012) W. A. Beltran et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Dystrophin Isoform Induction In Vivo by Antisense-mediated Alternative Splicing
- (2010) Sue Fletcher 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
- Efficacy of systemic morpholino exon-skipping in duchenne dystrophy dogs
- (2009) Toshifumi Yokota et al. ANNALS OF NEUROLOGY
- Octa-guanidine Morpholino Restores Dystrophin Expression in Cardiac and Skeletal Muscles and Ameliorates Pathology in Dystrophic mdx Mice
- (2009) Bo Wu et al. MOLECULAR THERAPY
- Sustained Dystrophin Expression Induced by Peptide-conjugated Morpholino Oligomers in the Muscles of mdx Mice
- (2008) Natee Jearawiriyapaisarn et al. MOLECULAR THERAPY
- Systemic Microdystrophin Gene Delivery Improves Skeletal Muscle Structure and Function in Old Dystrophic mdx Mice
- (2008) Paul Gregorevic et al. MOLECULAR THERAPY
Find Funding. Review Successful Grants.
Explore over 25,000 new funding opportunities and over 6,000,000 successful grants.
ExploreCreate your own webinar
Interested in hosting your own webinar? Check the schedule and propose your idea to the Peeref Content Team.
Create Now