Francesco Muntoni

Employment

• University College London

Education

Education history is unavailable.

Projects & Funding

Projects & funding information is unavailable.

Publications (48)

• PDGFRα-directed fibroblast-homing peptides for targeted delivery of nucleic acid therapeutics
  2026 DOI: 10.64898/2026.10.19.759357
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• 289th ENMC international workshop: assessing and managing emerging AAV related toxicities after gene therapy for neuromuscular disorders, 26 - 28 September 2025, Hoofddorp, The Netherlands
  Neuromuscular Disorders 2026 DOI: 10.1016/j.nmd.2026.107371
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• From N-of-1 therapy to health-system implementation
  Nature Reviews Drug Discovery 2026 DOI: 10.1038/s41573-026-01538-9
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• Ten years of disease-modifying therapy in spinal muscular atrophy: lessons learned and future directions
  Nature Reviews Neurology 2026 DOI: 10.1038/s41582-026-01224-9
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• Matrix remodeling plays an etiological role in driving laminin-α2 deficient pathology
  2026 DOI: 10.64898/2026.06.28.735063
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• Descriptive characterization of ambulatory health states in Duchenne muscular dystrophy: Motor function trajectories and times to loss of ambulation
  Journal of Neuromuscular Diseases 2026 DOI: 10.1177/22143602251364694
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• Increased utrophin expression in healthy and DMD patient derived myoblasts in response to ERK1/2 and EZH2 inhibitor treatment
  2026 DOI: 10.64898/2026.04.13.718206
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• The unmet need of psychopharmacological intervention for neuropsychiatric and neurodevelopmental comorbidities in Duchenne muscular dystrophy: report of 5 workshops
  European Journal of Paediatric Neurology 2026 DOI: 10.1016/j.ejpn.2026.03.011
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• Functional Characterization of an Immortalized Human Muscle Cell Line Carrying the Heterozygous RyR1 p.T4288_A4290dup Associated With Rhabdomyolysis
  Human Mutation 2026 DOI: 10.1155/humu/8852225
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• Regional Expression of Dystrophin Gene Transcripts and Proteins in the Mouse Brain
  Konstantina Tetorou, Artadokht Aghaeipour, Shunyi Ma

  +11 more

  • Talia E. Gileadi
  • Amel Saoudi
  • Pablo Perdomo Quinteiro
  • Jorge Aragón
  • Maaike van Putten
  • +6 more
  Cells 2025 DOI: 10.3390/cells14181441
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• Muscle transcriptome profiling reveals novel molecular pathways and biomarkers in laminin-α2 deficient patients
  2025 DOI: 10.1101/2025.08.23.671372
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• A comprehensive framework for the interpretation of TTN missense variants
  Maria Francesca Di Feo, Martin Rees, Victoria Lillback +57 more
  2025 DOI: 10.1101/2025.07.17.25331109
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• Clinical applications of exon-skipping antisense oligonucleotides in neuromuscular diseases
  Molecular Therapy 2025 DOI: 10.1016/j.ymthe.2025.04.038
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• MRC Centre for Neuromuscular Diseases Biobank London: its role in the advancement of rare and neuromuscular diseases research
  Neuromuscular Disorders 2025 DOI: 10.1016/j.nmd.2025.105280
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• Duchenne muscular dystrophy: recent insights in brain related comorbidities
  Nature Communications 2025 DOI: 10.1038/s41467-025-56644-w
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• AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial
  Nature Medicine 2025 DOI: 10.1038/s41591-024-03304-z
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• Relationship between growth and ambulation loss in Duchenne muscular dystrophy boys on steroids
  Georgia Stimpson, Deborah A. Ridout, Anna Sárközy +4 more
  European Journal of Neurology 2024 DOI: 10.1111/ene.16415
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• AAV gene therapy for Duchenne Muscular Dystrophy: lessons learned from a phase 3 trial
  Giovanni Baranello, Francesco Muntoni A
  Gene Therapy 2024 DOI: 10.1038/s41434-024-00494-6
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• Strategies to improve the design of gapmer antisense oligonucleotide on allele-specific silencing
  Molecular Therapy - Nucleic Acids 2024 DOI: 10.1016/j.omtn.2024.102237
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• Understanding anxiety experienced by young males with Duchenne muscular dystrophy: a qualitative focus group study
  Neuromuscular Disorders 2024 DOI: 10.1016/j.nmd.2023.12.002
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