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Исследования: Muscle→IPF (18)
18 публикаций в теме «Muscle→IPF», проиндексированных PF-Atlas из PubMed и мировых журналов.
- Muscle satellite cell editing by LNP-CRISPR-Cas9 to resist muscle injury. · Cell reports · 2026
- Transgene-induced cardiotoxicity in high-dose AAV gene transfer. · Molecular therapy : the journal of the American Society of Gene Therapy · 2026
- Proteomics-based evaluation of AAV dystrophin gene therapy outcomes in mdx skeletal muscle. · JCI insight · 2026
- AAV9 gene therapy to target respiratory insufficiency in the D2.mdx mouse model of duchenne muscular dystrophy. · Respiratory physiology & neurobiology · 2026
- Enhancing AAV9-UFμDys1 Gene Therapy Efficacy Through Immunosuppression in Mice with Pre-Existing Immunity and Enabling Redosing Strategies for Duchenne Muscular Dystrophy. · Human gene therapy · 2026
- Quantitative Muscle Magnetic Resonance Outcomes in Patients With Duchenne Muscular Dystrophy: An Exploratory Analysis From the EMBARK Randomized Clinical Trial. · JAMA neurology · 2025
- All-in-one vectors for epigenetic CRISPR inhibition of DUX4-fl in facioscapulohumeral muscular dystrophy. · Molecular therapy. Methods & clinical development · 2025
- Dystrophin Restorative and Compensatory Gene Addition Therapies for Duchenne Muscular Dystrophy: Could CRISPRa Provide a Realistic Alternative? · Muscles (Basel, Switzerland) · 2025
- FAP-CAR-T cells reduce dystrophic muscle fibrosis, improving adeno-associated virus gene transfer efficacy. · Molecular therapy. Methods & clinical development · 2025
- Expression of full-length dystrophin reverses muscular dystrophy defects in young and old mdx4cv mice. · The Journal of clinical investigation · 2025
- The road toward AAV-mediated gene therapy of Duchenne muscular dystrophy. · Molecular therapy : the journal of the American Society of Gene Therapy · 2025
- Systemic delivery of AAV5, AAV8, and AAV9 packaging a C5-12-microdystrophin-FLAG expression cassette in non-human primates. · Molecular therapy. Methods & clinical development · 2025
- Adeno-Associated Virus 8 and 9 Myofibre Type/Size Tropism Profiling Reveals Therapeutic Effect of Microdystrophin in Canines. · Journal of cachexia, sarcopenia and muscle · 2025
- AAV-based TCAP delivery rescues mitochondria dislocation in limb-girdle muscular dystrophy R7. · Brain : a journal of neurology · 2025
- The Promise and Pitfalls of AAV-Mediated Gene Therapy for Duchenne Muscular Dystrophy. · Current issues in molecular biology · 2025
- Development of an AAV-delivered microRNA gene therapy for myotonic dystrophy type 1. · Molecular therapy : the journal of the American Society of Gene Therapy · 2025
- Engineering Targeted Gene Delivery Systems for Primary Hereditary Skeletal Myopathies: Current Strategies and Future Perspectives. · Biomedicines · 2025
- Fbxl3 deletion mitigates myopathy in mdx mice through upregulation of myogenin. · Biochemical and biophysical research communications · 2025
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