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Cell Death & Disease|May 12, 2026
Emerin is necessary for microtubule-organizing center translocation to the nuclear envelope of muscle cellsElisabetta Mattioli, Vittoria Cenni, Patrizia Sabatelli, 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.
Molecular Therapy : the Journal of the American Society of Gene Therapy|March 6, 2018
Efficient Non-viral Gene Delivery into Human Hematopoietic Stem Cells by Minicircle Sleeping Beauty Transposon VectorsMarta Holstein, Cristina Mesa-Nuñez, Csaba Miskey, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|July 14, 2011
Correction of murine SCID-X1 by lentiviral gene therapy using a codon-optimized IL2RG gene and minimal pretransplant conditioningMarshall W Huston, Niek P van Til, Trudi P Visser, et al.
Archives of Disease in Childhood|November 12, 2020
Clinical management of sickle cell liver disease in children and young adultsEirini Kyrana, David Rees, Florence Lacaille, et al.
Cell Reports|March 15, 2018
Bimodal CD40/Fas-Dependent Crosstalk between iNKT Cells and Tumor-Associated Macrophages Impairs Prostate Cancer ProgressionFilippo Cortesi, Gloria Delfanti, Andrea Grilli, et al.
Biochimica Et Biophysica Acta. Gene Regulatory Mechanisms|September 5, 2017
Transcriptional and epigenetic analyses of the DMD locus reveal novel cis‑acting DNA elements that govern muscle dystrophin expressionSamuele Gherardi, Matteo Bovolenta, Chiara Passarelli, et al.
Blood|July 19, 2017
Gene therapy for Wiskott-Aldrich syndrome in a severely affected adultEmma C Morris, Thomas Fox, Ronjon Chakraverty, et al.
Theranostics|February 28, 2019
MSC-Delivered Soluble TRAIL and Paclitaxel as Novel Combinatory Treatment for Pancreatic AdenocarcinomaFilippo Rossignoli, Carlotta Spano, Giulia Grisendi, et al.
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