We thank N. Garcia for initial care and discussions on the patients; M. David, L. Delestre, P. Dessen and D. Farabos for excellent technical assistance; A. Boutron and E. Lebigot for providing primary fibroblasts; P. Rustin , G. Pierron and R. J. Morscher for discussions; A. Paci and J.
The funders had no role in study design, data collection and analysis, preparation of the manuscript or decision to publish.Present address: Auron Therapeutics, Cambridge, MA, USAThese authors contributed equally: Chris Ottolenghi, Stéphane De Botton.
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