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Proceedings of the National Academy of Sciences of the United States of America|May 3, 2020
Acoustofluidic sonoporation for gene delivery to human hematopoietic stem and progenitor cellsJason N Belling, Liv K Heidenreich, Zhenhua Tian, et al.The New England Journal of Medicine|October 5, 2017
Hematopoietic Stem-Cell Gene Therapy for Cerebral AdrenoleukodystrophyFlorian Eichler, Christine Duncan, Patricia L Musolino, et al.Journal of the National Comprehensive Cancer Network : JNCCN|February 12, 2025
Pediatric Acute Lymphoblastic Leukemia, Version 2.2025, NCCN Clinical Practice Guidelines In OncologyHiroto Inaba, David Teachey, Colleen Annesley, et al.The New England Journal of Medicine|October 9, 2024
Lentiviral Gene Therapy for Cerebral AdrenoleukodystrophyFlorian Eichler, Christine N Duncan, Patricia L Musolino, et al.Blood|June 3, 2026
Long-term stability of posttranscriptional genetic silencing of BCL11A using a shmiR vector in Sickle Cell DiseaseErica B Esrick, Leslie Lehmann, Amy Federico, et al.The New England Journal of Medicine|April 30, 2025
Lentiviral Gene Therapy for Severe Leukocyte Adhesion Deficiency Type 1Claire Booth, Julián Sevilla, Elena Almarza, et al.Obstetrical & Gynecological Survey|December 30, 2025
Lentiviral Gene Therapy for Severe Leukocyte Adhesion Deficiency Type 1Claire Booth, Julián Sevilla, Elena Almarza, et al.The New England Journal of Medicine|October 15, 2025
Long-Term Safety and Efficacy of Gene Therapy for Adenosine Deaminase DeficiencyClaire Booth, Katelyn Masiuk, Konstantinos Vazouras, et al.Human Gene Therapy|April 30, 2014
Gene therapy: charting a future course--summary of a National Institutes of Health Workshop, April 12, 2013Marina O'Reilly, Howard J Federoff, Yuman Fong, et al.Blood|May 11, 2021
Long-term outcomes after gene therapy for adenosine deaminase severe combined immune deficiencyBryanna Reinhardt, Omar Habib, Kit L Shaw, et al.Pageof 3