Author [ Title] Type Year
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Adenosine Deaminase (ADA)-Deficient Severe Combined Immune Deficiency (SCID) in the US Immunodeficiency Network (USIDNet) Registry. J Clin Immunol. 2020..
Analyzing CRISPR genome-editing experiments with CRISPResso. Nat Biotechnol. 2016;34(7):695-697..
Antiviral drug screen identifies DNA-damage response inhibitor as potent blocker of SARS-CoV-2 replication. Cell Rep. 2021:108940.
AT1R Activating Autoantibodies in Hematopoietic Stem Cell Transplantation. Biol Blood Marrow Transplant. 2020..
Busulfan Pharmacokinetics in ADA SCID Gene Therapy. Biol Blood Marrow Transplant. 2020.
Consensus approach for the management of severe combined immune deficiency caused by adenosine deaminase deficiency. J Allergy Clin Immunol. 2018..
Diverse Approaches to Gene Therapy of Sickle Cell Disease. Annu Rev Med. 2022..
Eliminating SCID row: new approaches to SCID. Hematology Am Soc Hematol Educ Program. 2014;2014(1):475-480..
Gene therapy for blood diseases. Curr Opin Biotechnol. 2018;60:39-45..
Improving Gene Editing Outcomes in Human Hematopoietic Stem and Progenitor Cells by Temporal Control of DNA Repair. Stem Cells. 2018.
Potentially Therapeutic Levels of Anti-Sickling Globin Gene Expression Following Lentivirus-mediated Gene Transfer in Sickle Cell Disease Bone Marrow CD34(+)Cells. Exp Hematol. 2015.
Primary Immune Deficiency Treatment Consortium (PIDTC) update. J Allergy Clin Immunol. 2016..
Prostate cancer originating in basal cells progresses to adenocarcinoma propagated by luminal-like cells. Proc Natl Acad Sci U S A. 2013.