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Molecular Therapy : the Journal of the American Society of Gene Therapy|July 31, 2025
Treatment of GATA2 deficiency by allele-specific CRISPR-Cas9-directed gene correction in hematopoietic stem cellsThomas Wisbech Skov, Jonas Holst Wolff, Didde Haslund, et al.
Iscience|May 28, 2019
CRISPR/Cas9 Genome Engineering in Engraftable Human Brain-Derived Neural Stem CellsDaniel P Dever, Samantha G Scharenberg, Joab Camarena, et al.
Leukemia|March 16, 2023
Impact of U2AF1 mutations on circular RNA expression in myelodysplastic neoplasmsEileen Wedge, Ulvi Ahmadov, Thomas B Hansen, et al.
Science (New York, N.Y.)|March 5, 2016
Activation of proto-oncogenes by disruption of chromosome neighborhoodsDenes Hnisz, Abraham S Weintraub, Daniel S Day, et al.
Molecular Therapy. Advances|July 13, 2026
AAV vector production in suspension cells using PEI transfection and sodium butyrate with orthogonal assessment of function and qualitySujan Ravendran, Simon Fammé, Maya Graham Noer, et al.
Cell Reports|March 12, 2025
Distinctive CD8<sup>+</sup> T cell activation by antigen-presenting plasmacytoid dendritic cells compared to conventional dendritic cellsRenée M van der Sluis, Juan L García-Rodríguez, Ian Helstrup Nielsen, et al.
Cell Stem Cell|May 4, 2019
Highly Efficient and Marker-free Genome Editing of Human Pluripotent Stem Cells by CRISPR-Cas9 RNP and AAV6 Donor-Mediated Homologous RecombinationRenata M Martin, Kazuya Ikeda, M Kyle Cromer, 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.
Nature Medicine|March 19, 2021
Gene replacement of α-globin with β-globin restores hemoglobin balance in β-thalassemia-derived hematopoietic stem and progenitor cellsM Kyle Cromer, Joab Camarena, Renata M Martin, et al.
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