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Cell|March 26, 2013
Single-cell dynamics of genome-nuclear lamina interactionsJop Kind, Ludo Pagie, Havva Ortabozkoyun, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|November 7, 2007
Generation of potent and stable human CD4+ T regulatory cells by activation-independent expression of FOXP3Sarah E Allan, Alicia N Alstad, Natacha Merindol, 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.
Molecular Therapy : the Journal of the American Society of Gene Therapy|March 25, 2026
CRISPR-Cas9-Mediated Upregulation of Utrophin Ameliorates Duchenne Muscular DystrophyMaëlle Ralu, Simon Guiraud, Sumitava Dastidar, et al.
Molecular Therapy. Nucleic Acids|April 24, 2023
Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategiesMégane Brusson, Anne Chalumeau, Pierre Martinucci, et al.
Science Translational Medicine|November 19, 2010
Identification of hematopoietic stem cell-specific miRNAs enables gene therapy of globoid cell leukodystrophyBernhard Gentner, Ilaria Visigalli, Hidefumi Hiramatsu, et al.
Blood Advances|February 26, 2021
Correction of β-thalassemia by CRISPR/Cas9 editing of the α-globin locus in human hematopoietic stem cellsGiulia Pavani, Anna Fabiano, Marine Laurent, 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.
Molecular Therapy : the Journal of the American Society of Gene Therapy|November 15, 2018
Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic RearrangementsAnnalisa Lattanzi, Vasco Meneghini, Giulia Pavani, et al.
Human Gene Therapy|September 8, 2021
Recent Progress in Genome Editing for Gene Therapy Applications: The French PerspectiveMario Amendola, Aurélie Bedel, Ana Buj-Bello, et al.
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