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American Journal of Human Genetics|September 6, 2023
High-throughput transcriptome analyses from ASPIRO, a phase 1/2/3 study of gene replacement therapy for X-linked myotubular myopathyGaia Andreoletti, Oriana Romano, Hsin-Jung Chou, et al.
Molecular Therapy. Nucleic Acids|January 30, 2017
Reactivating Fetal Hemoglobin Expression in Human Adult Erythroblasts Through BCL11A Knockdown Using Targeted EndonucleasesCarmen F Bjurström, Michelle Mojadidi, John Phillips, et al.
Experimental Hematology|February 15, 2015
Potentially therapeutic levels of anti-sickling globin gene expression following lentivirus-mediated gene transfer in sickle cell disease bone marrow CD34+ cellsFabrizia Urbinati, Phillip W Hargrove, Sabine Geiger, et al.
Proceedings of the National Academy of Sciences of the United States of America|January 25, 2006
Retroviral vector integration deregulates gene expression but has no consequence on the biology and function of transplanted T cellsAlessandra Recchia, Chiara Bonini, Zulma Magnani, et al.
Molecular Therapy. Methods & Clinical Development|June 2, 2015
The human ankyrin 1 promoter insulator sustains gene expression in a β-globin lentiviral vector in hematopoietic stem cellsZulema Romero, Beatriz Campo-Fernandez, Jennifer Wherley, et al.
Cytotherapy|July 23, 2017
Preclinical studies for a phase 1 clinical trial of autologous hematopoietic stem cell gene therapy for sickle cell diseaseFabrizia Urbinati, Jennifer Wherley, Sabine Geiger, et al.
The Journal of Clinical Investigation|August 3, 2007
Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapyAlessandro Aiuti, Barbara Cassani, Grazia Andolfi, et al.
Science Translational Medicine|October 14, 2016
Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cellsMark A DeWitt, Wendy Magis, Nicolas L Bray, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|July 14, 2016
CRISPR/Cas9-Mediated Correction of the Sickle Mutation in Human CD34+ cellsMegan D Hoban, Dianne Lumaquin, Caroline Y Kuo, et al.
Molecular Therapy. Methods & Clinical Development|February 14, 2024
AAV8 gene therapy reverses cardiac pathology and prevents early mortality in a mouse model of Friedreich's ataxiaJoshua C Chang, Molly R Ryan, Marie C Stark, et al.
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