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Molecular Therapy. Methods & Clinical Development|March 25, 2025
Highly efficient <i>in vivo</i> hematopoietic stem cell transduction using an optimized self-complementary adeno-associated virusCarsten T Charlesworth, Shota Homma, Anais K Amaya, et al.
Nature Biotechnology|August 3, 2023
High-efficiency transgene integration by homology-directed repair in human primary cells using DNA-PKcs inhibitionSridhar Selvaraj, William N Feist, Sebastien Viel, et al.
Biorxiv : the Preprint Server for Biology|June 4, 2025
<i>In utero</i> lipid nanoparticle delivery achieves robust editing in hematopoietic stem cellsAtesh K Worthington, Beltran Borges, Tony Lum, et al.
Proceedings of the National Academy of Sciences of the United States of America|March 20, 2015
Neomorphic effects of recurrent somatic mutations in Yin Yang 1 in insulin-producing adenomasM Kyle Cromer, Murim Choi, Carol Nelson-Williams, et al.
Biorxiv : the Preprint Server for Biology|April 27, 2026
Combinatorial base editing couples disease correction with lineage amplification in hematopoietic stem and progenitor cellsKun Jia, Eric Soupene, Roshani Sinha, et al.
Cell Reports. Medicine|September 18, 2025
Lentiviral vectors for hematopoietic stem cell gene therapy restore α-globin expression in α-thalassemia red blood cellsEva E R Segura, Kevyn Hart, Beatriz Campo Fernandez, et al.
Journal of Pediatric Gastroenterology and Nutrition|January 31, 2023
Fecal Microbiota Transplantation for Clostridioides difficile Infection in Immunocompromised Pediatric PatientsKatie R Conover, Imad Absah, Sonia Ballal, et al.
Hepatology (Baltimore, Md.)|March 10, 2022
Risk of variceal hemorrhage and pretransplant mortality in children with biliary atresiaLee M Bass, Wen Ye, Kieran Hawthorne, et al.
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