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Frontiers in Immunology|January 23, 2023
CRISPR-Cas9-AAV versus lentivector transduction for genome modification of X-linked severe combined immunodeficiency hematopoietic stem cellsJulie Brault, Taylor Liu, Siyuan Liu, et al.
Nature Medicine|August 8, 2018
A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cellsChristopher A Vakulskas, Daniel P Dever, Garrett R Rettig, et al.
Science Translational Medicine|June 17, 2021
Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell diseaseAnnalisa Lattanzi, Joab Camarena, Premanjali Lahiri, et al.
Cell Stem Cell|September 29, 2020
Loss of Extreme Long-Range Enhancers in Human Neural Crest Drives a Craniofacial DisorderHannah K Long, Marco Osterwalder, Ian C Welsh, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|April 1, 2021
Targeted replacement of full-length CFTR in human airway stem cells by CRISPR-Cas9 for pan-mutation correction in the endogenous locusSriram Vaidyanathan, Ron Baik, Lu Chen, et al.
Blood|December 6, 2019
Myeloid cell-targeted miR-146a mimic inhibits NF-κB-driven inflammation and leukemia progression in vivoYu-Lin Su, Xiuli Wang, Mati Mann, et al.
Nature Methods|February 1, 2019
T cell antigen discovery via trogocytosisGuideng Li, Michael T Bethune, Stephanie Wong, et al.
Cell|March 18, 2014
Heme-mediated SPI-C induction promotes monocyte differentiation into iron-recycling macrophagesMalay Haldar, Masako Kohyama, Alex Yick-Lun So, et al.
Cell Reports|January 4, 2025
Dual α-globin-truncated erythropoietin receptor knockin restores hemoglobin production in α-thalassemia-derived erythroid cellsSimon N Chu, Eric Soupene, Devesh Sharma, et al.
Molecular Therapy. Nucleic Acids|January 6, 2016
TALENs Facilitate Single-step Seamless SDF Correction of F508del CFTR in Airway Epithelial Submucosal Gland Cell-derived CF-iPSCsShingo Suzuki, R Geoffrey Sargent, Beate Illek, et al.
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