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Molecular Therapy. Methods & Clinical Development|August 25, 2018
An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease PhenotypeLeslie Weber, Valentina Poletti, Elisa Magrin, et al.
Stem Cell Reports|June 20, 2025
Telmisartan is neuroprotective in a hiPSC-derived spinal microtissue model for C9orf72 ALS via inhibition of neuroinflammationBerkiye Sonustun, Björn F Vahsen, Mario Ledesma-Terrón, 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.
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.
Haematologica|February 24, 2018
Plerixafor enables safe, rapid, efficient mobilization of hematopoietic stem cells in sickle cell disease patients after exchange transfusionChantal Lagresle-Peyrou, François Lefrère, Elisa Magrin, et al.
Elife|March 14, 2025
A microglia clonal inflammatory disorder in Alzheimer's diseaseRocio Vicario, Stamatina Fragkogianni, Leslie Weber, et al.
The New England Journal of Medicine|March 2, 2017
Gene Therapy in a Patient with Sickle Cell DiseaseJean-Antoine Ribeil, Salima Hacein-Bey-Abina, Emmanuel Payen, et al.
Biorxiv : the Preprint Server for Biology|February 8, 2024
A microglia clonal inflammatory disorder in Alzheimer's DiseaseRocio Vicario, Stamatina Fragkogianni, Leslie Weber, et al.
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