Evaluating the potential of novel genetic approaches for the treatment of Duchenne muscular dystrophy
出版年份 2021 全文链接
标题
Evaluating the potential of novel genetic approaches for the treatment of Duchenne muscular dystrophy
作者
关键词
-
出版物
EUROPEAN JOURNAL OF HUMAN GENETICS
Volume -, Issue -, Pages -
出版商
Springer Science and Business Media LLC
发表日期
2021-02-10
DOI
10.1038/s41431-021-00811-2
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。- TCTEX1D1 is a genetic modifier of disease progression in Duchenne muscular dystrophy
- (2020) Pietro Spitali et al. EUROPEAN JOURNAL OF HUMAN GENETICS
- Duchenne muscular dystrophy animal models for high-throughput drug discovery and precision medicine
- (2020) Nalinda B. Wasala et al. Expert Opinion on Drug Discovery
- Integrating gene delivery and gene-editing technologies by adenoviral vector transfer of optimized CRISPR-Cas9 components
- (2020) Ignazio Maggio et al. GENE THERAPY
- Single SERCA2a Therapy Ameliorated Dilated Cardiomyopathy for 18 Months in a Mouse Model of Duchenne Muscular Dystrophy
- (2020) Nalinda B. Wasala et al. MOLECULAR THERAPY
- AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer
- (2020) Helena Costa Verdera et al. MOLECULAR THERAPY
- Somatic gene editing ameliorates skeletal and cardiac muscle failure in pig and human models of Duchenne muscular dystrophy
- (2020) A. Moretti et al. NATURE MEDICINE
- Engineering adeno-associated virus vectors for gene therapy
- (2020) Chengwen Li et al. NATURE REVIEWS GENETICS
- CRISPR-engineered T cells in patients with refractory cancer
- (2020) Edward A. Stadtmauer et al. SCIENCE
- Enhanced CRISPR-Cas9 correction of Duchenne muscular dystrophy in mice by a self-complementary AAV delivery system
- (2020) Yu Zhang et al. Science Advances
- rAAVrh74.MCK.GALGT2 Protects Against Loss of Hemodynamic Function in the Aging mdx Mouse Heart
- (2019) Rui Xu et al. MOLECULAR THERAPY
- CRISPR-Cas9 corrects Duchenne muscular dystrophy exon 44 deletion mutations in mice and human cells
- (2019) Yi-Li Min et al. Science Advances
- In vivo RNA editing of point mutations via RNA-guided adenosine deaminases
- (2019) Dhruva Katrekar et al. NATURE METHODS
- Identification of preexisting adaptive immunity to Cas9 proteins in humans
- (2019) Carsten T. Charlesworth et al. NATURE MEDICINE
- Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy
- (2019) Christopher E. Nelson et al. NATURE MEDICINE
- Development of Novel Micro-dystrophins with Enhanced Functionality
- (2019) Julian N. Ramos et al. MOLECULAR THERAPY
- The potential of utrophin and dystrophin combination therapies for Duchenne muscular dystrophy
- (2019) Simon Guiraud et al. HUMAN MOLECULAR GENETICS
- Therapeutic developments for Duchenne muscular dystrophy
- (2019) Ingrid E. C. Verhaart et al. Nature Reviews Neurology
- Unified energetics analysis unravels SpCas9 cleavage activity for optimal gRNA design
- (2019) Dong Zhang et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Maximizing the Benefit of Life-Saving Treatments for Pompe Disease, Spinal Muscular Atrophy, and Duchenne Muscular Dystrophy Through Newborn Screening
- (2019) Mei Baker et al. JAMA Neurology
- AAV9 Edits Muscle Stem Cells in Normal and Dystrophic Adult Mice
- (2019) Michael E. Nance et al. MOLECULAR THERAPY
- Exons 45-55 skipping using mutation-tailored cocktails of antisense morpholinos in the DMD gene
- (2019) Yusuke Echigoya et al. MOLECULAR THERAPY
- A cytosine deaminase for programmable single-base RNA editing
- (2019) Omar O. Abudayyeh et al. SCIENCE
- Peptide-conjugate antisense based splice-correction for Duchenne muscular dystrophy and other neuromuscular diseases
- (2019) Maria K. Tsoumpra et al. EBioMedicine
- Non-immunogenic utrophin gene therapy for the treatment of muscular dystrophy animal models
- (2019) Yafeng Song et al. NATURE MEDICINE
- Surrogate gene therapy for muscular dystrophy
- (2019) Kay E. Davies et al. NATURE MEDICINE
- CRISPR RNAs trigger innate immune responses in human cells
- (2018) Sojung Kim et al. GENOME RESEARCH
- Exon-skipping advances for Duchenne muscular dystrophy
- (2018) Lucía Echevarría et al. HUMAN MOLECULAR GENETICS
- Cyclic Peptides to Improve Delivery and Exon Skipping of Antisense Oligonucleotides in a Mouse Model for Duchenne Muscular Dystrophy
- (2018) Silvana M.G. Jirka et al. MOLECULAR THERAPY
- Efficient and precise editing of endogenous transcripts with SNAP-tagged ADARs
- (2018) Paul Vogel et al. NATURE METHODS
- Antisense PMO cocktails effectively skip dystrophin exons 45-55 in myotubes transdifferentiated from DMD patient fibroblasts
- (2018) Joshua Lee et al. PLoS One
- Tracking disease progression non-invasively in Duchenne and Becker muscular dystrophies
- (2018) Pietro Spitali et al. Journal of Cachexia Sarcopenia and Muscle
- Combined Therapies for Duchenne Muscular Dystrophy to Optimize Treatment Efficacy
- (2018) Gonzalo Cordova et al. Frontiers in Genetics
- Personalized gene and cell therapy for Duchenne Muscular Dystrophy
- (2018) Florian Barthélémy et al. NEUROMUSCULAR DISORDERS
- Efficacy of Multi-exon Skipping Treatment in Duchenne Muscular Dystrophy Dog Model Neonates
- (2018) Kenji Rowel Q. Lim et al. MOLECULAR THERAPY
- High prevalence of Streptococcus pyogenes Cas9-reactive T cells within the adult human population
- (2018) Dimitrios L. Wagner et al. NATURE MEDICINE
- 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
- Reducing sarcolipin expression mitigates Duchenne muscular dystrophy and associated cardiomyopathy in mice
- (2017) Antanina Voit 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
- Eteplirsen in the treatment of Duchenne muscular dystrophy
- (2017) Kenji Rowel Lim et al. Drug Design Development and Therapy
- Development of Exon Skipping Therapies for Duchenne Muscular Dystrophy: A Critical Review and a Perspective on the Outstanding Issues
- (2017) Annemieke Aartsma-Rus et al. Nucleic Acid Therapeutics
- Efficacy and Safety Profile of Tricyclo-DNA Antisense Oligonucleotides in Duchenne Muscular Dystrophy Mouse Model
- (2017) Karima Relizani et al. Molecular Therapy-Nucleic Acids
- Nanoparticle delivery of Cas9 ribonucleoprotein and donor DNA in vivo induces homology-directed DNA repair
- (2017) Kunwoo Lee et al. Nature Biomedical Engineering
- Lentiviral vectors can be used for full-length dystrophin gene therapy
- (2017) John R. Counsell et al. Scientific Reports
- Cognitive dysfunction in Duchenne muscular dystrophy: a possible role for neuromodulatory immune molecules
- (2016) Mark G. Rae et al. JOURNAL OF NEUROPHYSIOLOGY
- The Pathogenesis and Therapy of Muscular Dystrophies
- (2015) Simon Guiraud et al. Annual Review of Genomics and Human Genetics
- In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy
- (2015) C. E. Nelson et al. SCIENCE
- Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy
- (2015) David G. Ousterout et al. Nature Communications
- What Can We Learn From Clinical Trials of Exon Skipping for DMD?
- (2014) Qi-long Lu et al. Molecular Therapy-Nucleic Acids
- Clinical Trials Using Antisense Oligonucleotides in Duchenne Muscular Dystrophy
- (2013) Taeyoung Koo et al. HUMAN GENE THERAPY
- 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
Discover Peeref hubs
Discuss science. Find collaborators. Network.
Join a conversationPublish scientific posters with Peeref
Peeref publishes scientific posters from all research disciplines. Our Diamond Open Access policy means free access to content and no publication fees for authors.
Learn More