検索結果 - Muntoni, Francesco

Disorders of Voluntary Muscle, 著者: Muntoni, Francesco

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The authors reply: Letter on: "Myostatin inhibition in combination with antisense oligonucleotide therapy improves outcomes in spinal muscular atrophy" by Zhou et al. 著者: Zhou, Haiyan, Muntoni, Francesco

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Changes in Myonuclear Number During Postnatal Growth – Implications for AAV Gene Therapy for Muscular Dystrophy 著者: Morgan, Jennifer, Muntoni, Francesco

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Muscular dystrophies due to glycosylation defects 著者: Muntoni, Francesco, Torelli, Silvia, Brockington, Martin

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CD133+ cells derived from skeletal muscles of Duchenne muscular dystrophy patients have a compromised myogenic and muscle regenerative capability 著者: Meng, Jinhong, Muntoni, Francesco, Morgan, Jennifer

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Defining and identifying satellite cell-opathies within muscular dystrophies and myopathies 著者: Ganassi, Massimo, Muntoni, Francesco, Zammit, Peter S.

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Are Human and Mouse Satellite Cells Really the Same? 著者: Boldrin, Luisa, Muntoni, Francesco, Morgan, Jennifer E.

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Generation of two genomic-integration-free DMD iPSC lines with mutations affecting all dystrophin isoforms and potentially amenable to exon-skipping 著者: Ferrari, Giulia, Muntoni, Francesco, Tedesco, Francesco Saverio

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Considerations for Systemic Use of Gene Therapy 著者: Byrne, Barry J., Corti, Manuela, Muntoni, Francesco

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Gapmer Antisense Oligonucleotides Suppress the Mutant Allele of COL6A3 and Restore Functional Protein in Ullrich Muscular Dystrophy 著者: Marrosu, Elena, Ala, Pierpaolo, Muntoni, Francesco, Zhou, Haiyan

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Non‐sarcolemmal Muscular Dystrophies 著者: Brown, Susan C., Muntoni, Francesco, Sewry, Caroline A.

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Clinical utility gene card for: Multi-minicore disease 著者: Lillis, Suzanne, Abbs, Steve, Ferreiro, Ana, Muntoni, Francesco, Jungbluth, Heinz

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Mature Adult Dystrophic Mouse Muscle Environment Does Not Impede Efficient Engrafted Satellite Cell Regeneration and Self-Renewal 著者: Boldrin, Luisa, Zammit, Peter Steven, Muntoni, Francesco, Morgan, Jennifer Elizabeth

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Clinical utility gene card for: Central core disease 著者: Lillis, Suzanne, Abbs, Stephen, Mueller, Clemens R, Muntoni, Francesco, Jungbluth, Heinz

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Donor Satellite Cell Engraftment is Significantly Augmented When the Host Niche is Preserved and Endogenous Satellite Cells are Incapacitated 著者: Boldrin, Luisa, Neal, Alice, Zammit, Peter S, Muntoni, Francesco, Morgan, Jennifer E

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The effect of the muscle environment on the regenerative capacity of human skeletal muscle stem cells 著者: Meng, Jinhong, Bencze, Maximilien, Asfahani, Rowan, Muntoni, Francesco, Morgan, Jennifer E

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Genome Editing and Muscle Stem Cells as a Therapeutic Tool for Muscular Dystrophies 著者: Pini, Veronica, Morgan, Jennifer E., Muntoni, Francesco, O’Neill, Helen C.

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Antisense Oligonucleotide-Based Therapy for Neuromuscular Disease 著者: Sardone, Valentina, Zhou, Haiyan, Muntoni, Francesco, Ferlini, Alessandra, Falzarano, Maria Sofia

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Human Skeletal Muscle–derived CD133(+) Cells Form Functional Satellite Cells After Intramuscular Transplantation in Immunodeficient Host Mice 著者: Meng, Jinhong, Chun, Soyon, Asfahani, Rowan, Lochmüller, Hanns, Muntoni, Francesco, Morgan, Jennifer

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Gene therapy in Duchenne muscular dystrophy: Identifying and preparing for the challenges ahead 著者: Heslop, Emma, Turner, Cathy, Irvin, Anna, Muntoni, Francesco, Straub, Volker, Guglieri, Michela

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