CRISPR/Cas9-mediated correction of the sickle mutation in human CD34+ cells MD Hoban, D Lumaquin, CY Kuo, Z Romero, J Long, M Ho, CS Young, ... Molecular Therapy 24 (9), 1561-1569, 2016 | 230 | 2016 |
Lentiviral gene therapy for X-linked chronic granulomatous disease DB Kohn, C Booth, EM Kang, SY Pai, KL Shaw, G Santilli, M Armant, ... Nature medicine 26 (2), 200-206, 2020 | 229 | 2020 |
Site-specific gene editing of human hematopoietic stem cells for X-linked hyper-IgM syndrome CY Kuo, JD Long, B Campo-Fernandez, S de Oliveira, AR Cooper, ... Cell reports 23 (9), 2606-2616, 2018 | 141 | 2018 |
Long-term outcomes of 176 patients with X-linked hyper-IgM syndrome treated with or without hematopoietic cell transplantation MT de la Morena, D Leonard, TR Torgerson, O Cabral-Marques, M Slatter, ... Journal of Allergy and Clinical Immunology 139 (4), 1282-1292, 2017 | 133 | 2017 |
Editing the sickle cell disease mutation in human hematopoietic stem cells: comparison of endonucleases and homologous donor templates Z Romero, A Lomova, S Said, A Miggelbrink, CY Kuo, ... Molecular therapy 27 (8), 1389-1406, 2019 | 101 | 2019 |
Gene therapy for the treatment of primary immune deficiencies CY Kuo, DB Kohn Current allergy and asthma reports 16, 1-8, 2016 | 66 | 2016 |
Reactivating fetal hemoglobin expression in human adult erythroblasts through BCL11A knockdown using targeted endonucleases CF Bjurström, M Mojadidi, J Phillips, C Kuo, S Lai, GR Lill, A Cooper, ... Molecular therapy-nucleic acids 5, 2016 | 62 | 2016 |
New frontiers in the therapy of primary immunodeficiency: from gene addition to gene editing DB Kohn, CY Kuo Journal of Allergy and Clinical Immunology 139 (3), 726-732, 2017 | 57 | 2017 |
The PTH/PTHrP-SIK3 pathway affects skeletogenesis through altered mTOR signaling F Csukasi, I Duran, M Barad, T Barta, I Gudernova, L Trantirek, JH Martin, ... Science Translational Medicine 10 (459), eaat9356, 2018 | 46 | 2018 |
Newborn screening for severe combined immunodeficiency does not identify bare lymphocyte syndrome CY Kuo, J Chase, MG Lloret, ER Stiehm, T Moore, MJM Aguilera, JL Siles, ... Journal of Allergy and Clinical Immunology 131 (6), 1693-1695, 2013 | 46 | 2013 |
Immune and genetic features of the chromosome 22q11. 2 deletion (DiGeorge syndrome) CY Kuo, R Signer, SC Saitta Current Allergy and Asthma Reports 18, 1-7, 2018 | 42 | 2018 |
Lentiviral gene therapy for artemis-deficient SCID MJ Cowan, J Yu, J Facchino, C Fraser-Browne, U Sanford, M Kawahara, ... New England Journal of Medicine 387 (25), 2344-2355, 2022 | 38 | 2022 |
Fiscal implications of newborn screening in the diagnosis of severe combined immunodeficiency C Kubiak, S Jyonouchi, C Kuo, M Garcia-Lloret, MJ Dorsey, J Sleasman, ... The Journal of Allergy and Clinical Immunology: In Practice 2 (6), 697-702, 2014 | 33 | 2014 |
Outcomes following treatment for ADA-deficient severe combined immunodeficiency: a report from the PIDTC GDE Cuvelier, BR Logan, SE Prockop, RH Buckley, CY Kuo, LM Griffith, ... Blood, The Journal of the American Society of Hematology 140 (7), 685-705, 2022 | 31 | 2022 |
Optimizing integration and expression of transgenic bruton's tyrosine kinase for CRISPR-Cas9-Mediated gene editing of X-linked agammaglobulinemia DH Gray, I Villegas, J Long, J Santos, A Keir, A Abele, CY Kuo, DB Kohn The CRISPR journal 4 (2), 191-206, 2021 | 27 | 2021 |
Characterization of gene alterations following editing of the β-globin gene locus in hematopoietic stem/progenitor cells J Long, MD Hoban, AR Cooper, ML Kaufman, CY Kuo, ... Molecular therapy 26 (2), 468-479, 2018 | 26 | 2018 |
Adenosine deaminase (ADA)–deficient severe combined immune deficiency (SCID) in the US Immunodeficiency Network (USIDNet) Registry CY Kuo, E Garabedian, J Puck, MJ Cowan, KE Sullivan, RH Buckley, ... Journal of clinical immunology 40, 1124-1131, 2020 | 25 | 2020 |
Profound T cell Lymphopenia associated with prenatal exposure to purine antagonists detected by TREC newborn screening CY Kuo, MI Garcia-Lloret, P Slev, JF Bohnsack, K Chen The journal of allergy and clinical immunology. In practice 5 (1), 198, 2017 | 23 | 2017 |
Novel compound heterozygote variations in FADD identified to cause FAS-associated protein with death domain deficiency LA Kohn, JD Long, EC Trope, CY Kuo Journal of Clinical Immunology 40, 658-661, 2020 | 16 | 2020 |
Long-term outcomes of 176 patients with X-linked hyper-IgM syndrome treated with or without hematopoietic cell transplantation M Morena, D Leonard, TR Torgerson, O Cabral-Marques, M Slatter, ... Journal of allergy and clinical immunology, 2017 | 16 | 2017 |