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

por Zhou, Haiyan, Muntoni, Francesco
Publicado 2020

Changes in Myonuclear Number During Postnatal Growth – Implications for AAV Gene Therapy for Muscular Dystrophy

por Morgan, Jennifer, Muntoni, Francesco
Publicado 2021

CD133+ cells derived from skeletal muscles of Duchenne muscular dystrophy patients have a compromised myogenic and muscle regenerative capability

por Meng, Jinhong, Muntoni, Francesco, Morgan, Jennifer
Publicado 2018

Defining and identifying satellite cell-opathies within muscular dystrophies and myopathies

por Ganassi, Massimo, Muntoni, Francesco, Zammit, Peter S.
Publicado 2022

Generation of two genomic-integration-free DMD iPSC lines with mutations affecting all dystrophin isoforms and potentially amenable to exon-skipping

por Ferrari, Giulia, Muntoni, Francesco, Tedesco, Francesco Saverio
Publicado 2020

Letter to the Editor: In response to P.R. Clemens et al., Efficacy and Safety of Viltolarsen in Boys with Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year...

por Muntoni, Francesco, Straub, Volker, Servais, Laurent, Mercuri, Eugenio
Publicado 2023

Gapmer Antisense Oligonucleotides Suppress the Mutant Allele of COL6A3 and Restore Functional Protein in Ullrich Muscular Dystrophy

por Marrosu, Elena, Ala, Pierpaolo, Muntoni, Francesco, Zhou, Haiyan
Publicado 2017

Lessons Learned From Translational Research in Neuromuscular Diseases: Impact on Study Design, Outcome Measures and Managing Expectation

por Stimpson, Georgia, Chesshyre, Mary, Baranello, Giovanni, Muntoni, Francesco
Publicado 2021

Revised Hammersmith Scale for spinal muscular atrophy: Inter and intra-rater reliability and agreement

por Ramsey, Danielle, Ramdharry, Gita, Scoto, Mariacristina, Muntoni, Francesco, Wallace, Amanda
Publicado 2022

Donor Satellite Cell Engraftment is Significantly Augmented When the Host Niche is Preserved and Endogenous Satellite Cells are Incapacitated

por Boldrin, Luisa, Neal, Alice, Zammit, Peter S, Muntoni, Francesco, Morgan, Jennifer E
Publicado 2012

The effect of the muscle environment on the regenerative capacity of human skeletal muscle stem cells

por Meng, Jinhong, Bencze, Maximilien, Asfahani, Rowan, Muntoni, Francesco, Morgan, Jennifer E
Publicado 2015

Genome Editing and Muscle Stem Cells as a Therapeutic Tool for Muscular Dystrophies

por Pini, Veronica, Morgan, Jennifer E., Muntoni, Francesco, O’Neill, Helen C.
Publicado 2017

Antisense Oligonucleotide-Based Therapy for Neuromuscular Disease

por Sardone, Valentina, Zhou, Haiyan, Muntoni, Francesco, Ferlini, Alessandra, Falzarano, Maria Sofia
Publicado 2017

Gene therapy in Duchenne muscular dystrophy: Identifying and preparing for the challenges ahead

por Heslop, Emma, Turner, Cathy, Irvin, Anna, Muntoni, Francesco, Straub, Volker, Guglieri, Michela
Publicado 2021

Optimized lentiviral vector to restore full-length dystrophin via a cell-mediated approach in a mouse model of Duchenne muscular dystrophy

por Meng, Jinhong, Moore, Marc, Counsell, John, Muntoni, Francesco, Popplewell, Linda, Morgan, Jennifer
Publicado 2022

Contribution of Human Muscle-Derived Cells to Skeletal Muscle Regeneration in Dystrophic Host Mice

por Meng, Jinhong, Adkin, Carl F., Xu, Shi-wen, Muntoni, Francesco, Morgan, Jennifer E.
Publicado 2011

LAMA2-Related Dystrophies: Clinical Phenotypes, Disease Biomarkers, and Clinical Trial Readiness

por Sarkozy, Anna, Foley, A. Reghan, Zambon, Alberto A., Bönnemann, Carsten G., Muntoni, Francesco
Publicado 2020

MyoD-induced reprogramming of human fibroblasts and urinary stem cells in vitro: protocols and their applications

por Rossi, Rachele, Torelli, Silvia, Ala, Pierpaolo, Weston, William, Morgan, Jennifer, Malhotra, Jyoti, Muntoni, Francesco
Publicado 2023

Altered Levels of MicroRNA-9, -206, and -132 in Spinal Muscular Atrophy and Their Response to Antisense Oligonucleotide Therapy

por Catapano, Francesco, Zaharieva, Irina, Scoto, Mariacristina, Marrosu, Elena, Morgan, Jennifer, Muntoni, Francesco, Zhou, Haiyan
Publicado 2016

Development of a novel startle response task in Duchenne muscular dystrophy

por Maresh, Kate, Papageorgiou, Andriani, Ridout, Deborah, Harrison, Neil, Mandy, William, Skuse, David, Muntoni, Francesco
Publicado 2022