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Molecular Therapy. Methods & Clinical Development|April 25, 2018
Preclinical Efficacy and Safety Evaluation of Hematopoietic Stem Cell Gene Therapy in a Mouse Model of MNGIERana Yadak, Raquel Cabrera-Pérez, Javier Torres-Torronteras, et al.Science Translational Medicine|December 15, 2015
Tracking genetically engineered lymphocytes long-term reveals the dynamics of T cell immunological memoryGiacomo Oliveira, Eliana Ruggiero, Maria Teresa Lupo Stanghellini, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|February 29, 2012
Preclinical safety and efficacy of human CD34(+) cells transduced with lentiviral vector for the treatment of Wiskott-Aldrich syndromeSamantha Scaramuzza, Luca Biasco, Anna Ripamonti, et al.Nature Medicine|March 27, 2019
Highly efficient therapeutic gene editing of human hematopoietic stem cellsYuxuan Wu, Jing Zeng, Benjamin P Roscoe, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|September 19, 2024
Preclinical lentiviral hematopoietic stem cell gene therapy corrects Pompe disease-related muscle and neurological manifestationsJohn K Yoon, Jeffrey W Schindler, Mariana Loperfido, et al.Nature Medicine|December 8, 2009
Comprehensive genomic access to vector integration in clinical gene therapyRichard Gabriel, Ralph Eckenberg, Anna Paruzynski, et al.Cell|May 1, 2026
Distinct in vivo dynamics of donor-derived stem cell memory CAR T cells post-allogeneic HSCT relapseLuca Gattinoni, Gabriele Inchingolo, Dennis C Harrer, et al.Nature Biotechnology|December 5, 2025
In vivo gene editing of human hematopoietic stem and progenitor cells using envelope-engineered virus-like particlesVladimir V Botchkarev, Sean Harrington, Matteo Stoppato, et al.Human Gene Therapy|July 25, 2024
Improving the Assessment of Risk Factors Relevant to Potential Carcinogenicity of Gene Therapies: A Consensus ArticleJan C Klapwijk, Alberto Del Rio Espinola, Silvana Libertini, et al.Science (New York, N.Y.)|July 13, 2013
Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophyAlessandra Biffi, Eugenio Montini, Laura Lorioli, et al.Pageof 4