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Molecular Therapy. Methods & Clinical Development|December 12, 2018
Pre-clinical Development of a Lentiviral Vector Expressing the Anti-sickling βAS3 Globin for Gene Therapy for Sickle Cell DiseaseValentina Poletti, Fabrizia Urbinati, Sabine Charrier, et al.
Plos One|December 23, 2014
Stochastic fluctuations and distributed control of gene expression impact cellular memoryGuillaume Corre, Daniel Stockholm, Ophélie Arnaud, et al.
Plos Biology|October 26, 2022
Global genome decompaction leads to stochastic activation of gene expression as a first step toward fate commitment in human hematopoietic cellsRomuald Parmentier, Laëtitia Racine, Alice Moussy, et al.
Cell Reports. Medicine|September 27, 2025
Multiplex base editing of BCL11A regulatory elements to treat sickle cell diseaseLetizia Fontana, Pierre Martinucci, Simone Amistadi, et al.
Disease Models & Mechanisms|January 8, 2026
A new dystrophin deficient rat model mirroring exon skipping in patients with DMD exon 45 deletionsTao Wang, Cynthia Daoud, Auriane Dubois, et al.
Blood|August 26, 2025
A prime editing strategy to rewrite the γ-globin promoters and reactivate fetal hemoglobin for sickle cell diseaseAnne Chalumeau, Maria Bou Dames, Letizia Fontana, et al.
Science Translational Medicine|November 29, 2019
Titin splicing regulates cardiotoxicity associated with calpain 3 gene therapy for limb-girdle muscular dystrophy type 2AWilliam Lostal, Carinne Roudaut, Marine Faivre, et al.
Communications Medicine|April 11, 2025
Rescue of lysosomal acid lipase deficiency in mice by rAAV8 liver gene transferMarine Laurent, Rim Harb, Christine Jenny, et al.
Scientific Reports|June 6, 2020
miR-379 links glucocorticoid treatment with mitochondrial response in Duchenne muscular dystrophyMathilde Sanson, Ai Vu Hong, Emmanuelle Massourides, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|February 4, 2024
An empowered, clinically viable hematopoietic stem cell gene therapy for the treatment of multisystemic mucopolysaccharidosis type IISabyasachi Das, Fatlum Rruga, Annita Montepeloso, et al.
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