Optogenetic modeling of human neuromuscular circuits in Duchenne muscular dystrophy with CRISPR and pharmacological corrections

Author:

Paredes-Redondo Amaia123ORCID,Harley Peter4ORCID,Maniati Eleni5,Ryan David6ORCID,Louzada Sandra6ORCID,Meng Jinhong7ORCID,Kowala Anna123ORCID,Fu Beiyuan6,Yang Fengtang6ORCID,Liu Pentao8ORCID,Marino Silvia1,Pourquié Olivier9ORCID,Muntoni Francesco710ORCID,Wang Jun5ORCID,Lieberam Ivo4ORCID,Lin Yung-Yao123ORCID

Affiliation:

1. Centre for Genomics and Child Health, Blizard Institute, Barts and the London School of Medicine and Dentistry, Queen Mary University of London, 4 Newark Street, London E1 2AT, UK.

2. Stem Cell Laboratory, National Bowel Research Centre, Blizard Institute, Barts and the London School of Medicine and Dentistry, Queen Mary University of London, 2 Newark Street, London E1 2AT, UK.

3. Centre for Predictive in vitro Model, Queen Mary University of London, Mile End Road, London E1 4NS, UK.

4. Centre for Stem Cells and Regenerative Medicine, MRC Centre for Neurodevelopmental Disorders, and Centre for Developmental Neurobiology, King’s College London, London, UK.

5. Centre for Cancer Genomics and Computational Biology, Barts Cancer Institute, Queen Mary University of London, London, UK.

6. Wellcome Sanger Institute, Wellcome Genome Campus, Hinxton, Cambridge CB10 1SA, UK.

7. UCL Great Ormond Street Institute of Child Health, 30 Guilford Street, London WC1N 1EH, UK.

8. School of Biomedical Sciences, Stem Cell and Regenerative Medicine Consortium, Li Ka Shing Faculty of Medicine, The University of Hong Kong, Hong Kong, China.

9. Department of Genetics and Department of Pathology, Brigham and Women’s Hospital, Harvard Medical School, 60 Fenwood Road, Boston, MA, USA.

10. NIHR Biomedical Research Centre, Great Ormond Street Hospital, Great Ormond Street, London, UK.

Abstract

SB-431542 restores neuromuscular synapses and motor function in a human microphysiological model of muscular dystrophy.

Publisher

American Association for the Advancement of Science (AAAS)

Subject

Multidisciplinary

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