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Communications Biology|March 7, 2024
SAM68 directs STING signaling to apoptosis in macrophagesDemi van der Horst, Naziia Kurmasheva, Mikkel H S Marqvorsen, et al.
Molecular Therapy. Methods & Clinical Development|September 6, 2021
Clinically relevant gene editing in hematopoietic stem cells for the treatment of pyruvate kinase deficiencySara Fañanas-Baquero, Oscar Quintana-Bustamante, Daniel P Dever, et al.
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.
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.
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.
Blood|February 24, 2021
Enhanced homology-directed repair for highly efficient gene editing in hematopoietic stem/progenitor cellsSuk See De Ravin, Julie Brault, Ronald J Meis, et al.
Blood Advances|December 14, 2023
Genetically corrected RAG2-SCID human hematopoietic stem cells restore V(D)J-recombinase and rescue lymphoid deficiencyMara Pavel-Dinu, Cameron L Gardner, Yusuke Nakauchi, et al.
The EMBO Journal|February 18, 2022
TLR2 and TLR7 mediate distinct immunopathological and antiviral plasmacytoid dendritic cell responses to SARS-CoV-2 infectionRenée M van der Sluis, Lamin B Cham, Albert Gris-Oliver, et al.
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