2021
Delville, M. et Al. « A combination of cyclophosphamide and interleukin-2 allows CD4+ T cells converted to Tregs to control scurfy syndrome » Blood 137, 2326-2336
Ramadier, S. et Al.
« Combination of lentiviral and genome editing technologies for the treatment of sickle cell disease »
Mol Ther
Amor, F. et Al.
« Cholesterol metabolism is a potential therapeutic target in Duchenne muscular dystrophy »
Journal of cachexia, sarcopenia and muscle 12, 677-693
Zhukouskaya, VV. et Al.
« A novel therapeutic strategy for skeletal disorders: Proof of concept of gene therapy for X-linked hypophosphatemia »
Science advances 7, eabj5018
Fouarge, E. et Al.
« Hierarchical Bayesian modelling of disease progression to inform clinical trial design in centronuclear myopathy »
Orphanet journal of rare diseases 16 (1), 3
