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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.Nature Neuroscience|February 24, 2009
Forebrain ependymal cells are Notch-dependent and generate neuroblasts and astrocytes after strokeMarie Carlén, Konstantinos Meletis, Christian Göritz, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|August 21, 2021
Combination of lentiviral and genome editing technologies for the treatment of sickle cell diseaseSophie Ramadier, Anne Chalumeau, Tristan Felix, et al.Cell|September 15, 2015
Genome-wide maps of nuclear lamina interactions in single human cellsJop Kind, Ludo Pagie, Sandra S de Vries, et al.Science Advances|September 12, 2020
Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotypeLeslie Weber, Giacomo Frati, Tristan Felix, et al.Cell|May 6, 2017
The Cohesin Release Factor WAPL Restricts Chromatin Loop ExtensionJudith H I Haarhuis, Robin H van der Weide, Vincent A Blomen, et al.Blood|March 10, 2018
Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locusChiara Antoniani, Vasco Meneghini, Annalisa Lattanzi, et al.Science Translational Medicine|November 26, 2025
Base editing of β0-thalassemia mutations as a therapeutic strategy for severe β-hemoglobinopathiesGiulia Hardouin, Pierre Martinucci, Samantha Scaramuzza, et al.Blood|February 5, 2021
A combination of cyclophosphamide and interleukin-2 allows CD4+ T cells converted to Tregs to control scurfy syndromeMarianne Delville, Florence Bellier, Juliette Leon, et al.Nature Communications|November 5, 2022
Base-editing-mediated dissection of a γ-globin cis-regulatory element for the therapeutic reactivation of fetal hemoglobin expressionPanagiotis Antoniou, Giulia Hardouin, Pierre Martinucci, et al.Pageof 4