Research ArticleGene Therapy

Titin splicing regulates cardiotoxicity associated with calpain 3 gene therapy for limb-girdle muscular dystrophy type 2A

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Science Translational Medicine  27 Nov 2019:
Vol. 11, Issue 520, eaat6072
DOI: 10.1126/scitranslmed.aat6072

Article Information

vol. 11 no. 520

PubMed: 
Print ISSN: 
Online ISSN: 
History: 
  • Received for publication January 30, 2019
  • Accepted for publication October 31, 2019
  • .

Author Information

  1. William Lostal1,
  2. Carinne Roudaut1,
  3. Marine Faivre1,
  4. Karine Charton1,*,
  5. Laurence Suel1,
  6. Nathalie Bourg1,
  7. Heather Best1,
  8. John Edward Smith2,
  9. Jochen Gohlke2,
  10. Guillaume Corre1,
  11. Xidan Li3,
  12. Zaher Elbeck3,
  13. Ralph Knöll3,4,
  14. Jack-Yves Deschamps5,
  15. Henk Granzier2 and
  16. Isabelle Richard1,
  1. 1Généthon INSERM, U951, INTEGRARE Research Unit, Evry F-91002, France.
  2. 2University of Arizona, Tucson, AZ 85724-5217, USA.
  3. 3Department of Medicine, Integrated Cardio Metabolic Centre (ICMC), Heart and Vascular Theme, Karolinska Institute, Stockholm SE-171 77, Sweden.
  4. 4Bioscience, Cardiovascular, Renal & Metabolism, BioPharmaceuticals R&D, AstraZeneca, Gothenburg, Sweden.
  5. 5Emergency and Critical Care Unit, ONIRIS, School of Veterinary Medicine, La Chantrerie, 44307 Nantes Cedex 03, France.
  1. Corresponding author. Email: richard{at}genethon.fr
  • * Present address: Erytech, 69008 Lyon, France.

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