Using patient iPSC-derived skeletal muscle models for development of a CRISPR-based exon removal therapeutic strategy

Daniel Moore (1) , Heather Steele-Stallard , Luca Pinton (2) , Valentina Lionello (3) , Salma Jalal , Jean-Marie Cuisset , Gisèle Bonne (4) , Peter S. Zammit (5) , Francesco Saverio Tedesco (6)

1 Princeton University
2 UCL - University College of London [London]
3 IGBMC - Institut de Génétique et de Biologie Moléculaire et Cellulaire
4 Centre de recherche en Myologie – U974 SU-INSERM
5 Randall Division of Cell and Molecular Biophysics
6 UCL - Department of Cell and Developmental Biology [London, UK]

Mots clés: LMNA gene; laminopathy; IPSC

Domaines: Génétique humaine; Organisation et fonctions cellulaires [q-bio.SC]

Daniel Moore, Heather Steele-Stallard, Luca Pinton, Valentina Lionello, Salma Jalal, et al.. Using patient iPSC-derived skeletal muscle models for development of a CRISPR-based exon removal therapeutic strategy. 4th International Meeting on Laminopathies, May 2023, Madrid, Spain. ⟨10.3233/JND-239001⟩. ⟨hal-04189588⟩