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Science Translational Medicine|October 16, 2024
High-fidelity PAMless base editing of hematopoietic stem cells to treat chronic granulomatous diseaseVera Bzhilyanskaya, Linyuan Ma, Siyuan Liu, et al.
Clinical Infectious Diseases : an Official Publication of the Infectious Diseases Society of America|November 18, 2017
The Changing Paradigm of Management of Liver Abscesses in Chronic Granulomatous DiseaseDavid M Straughan, Kaitlin C McLoughlin, John E Mullinax, 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.
The Journal of Clinical Investigation|August 10, 2023
A phase III randomized crossover trial of plerixafor versus G-CSF for treatment of WHIM syndromeDavid H McDermott, Daniel Velez, Elena Cho, et al.
Blood|June 4, 2021
CRISPR-targeted MAGT1 insertion restores XMEN patient hematopoietic stem cells and lymphocytesJulie Brault, Taylor Liu, Ezekiel Bello, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|April 27, 2019
Aberrant Clonal Hematopoiesis following Lentiviral Vector Transduction of HSPCs in a Rhesus MacaqueDiego A Espinoza, Xing Fan, Di Yang, et al.
The Journal of Allergy and Clinical Immunology|December 31, 2013
CXCR4/IgG-expressing plasma cells are associated with human gastrointestinal tissue inflammationClarisa M Buckner, Susan Moir, Lela Kardava, et al.
Nature Communications|June 28, 2022
Lentivector cryptic splicing mediates increase in CD34+ clones expressing truncated HMGA2 in human X-linked severe combined immunodeficiencySuk See De Ravin, Siyuan Liu, Colin L Sweeney, et al.
Nature Biotechnology|March 8, 2016
Targeted gene addition in human CD34(+) hematopoietic cells for correction of X-linked chronic granulomatous diseaseSuk See De Ravin, Andreas Reik, Pei-Qi Liu, et al.
Blood|May 22, 2010
Hypomorphic Rag mutations can cause destructive midline granulomatous diseaseSuk See De Ravin, Edward W Cowen, Kol A Zarember, et al.
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