Caroline Kuo, MD

Dr. Kuo is an Assistant Professor in Residence in the Division of Pediatric Allergy, Immunology, and Rheumatology at UCLA. Her clinical practice focuses on the diagnosis and treatment of patients with inborn errors of immunity. For the last 10 years, her research has focused on the development of site-specific gene editing approaches for the treatment of primary immune deficiencies, including X-linked Hyper IgM Syndrome, X-linked Agammaglobulinemia, and others using CRISPR/Cas9 technologies. Long-lasting immune correction of autologous hematopoietic stem cells in these diseases would not only allow immune reconstitution without the conditioning needed for traditional allogeneic stem cell transplant, but also restore immune surveillance and eliminate increased cancer risk seen in many diseases of the immune system.

1. Kubiak C, Jyonouchi S, Kuo C, Garcia-Lloret M, Dorsey M, Sleaseman J, Zbrozek A, Perez E. Fiscal Implications of Newborn Screening in the Diagnosis of Severe Combined Immunodeficiency. J Allergy Clin Immunol Pract. 2014 Nov-Dec;2(6):697-702.

2. Bjurstrom CF, Mojadidi M, Phillips J, Kuo C, Lai S, Lill GR, Cooper A, Kaufman M, Urbinati F, Wang X, Hollis RP, Kohn DB. Reactivating Fetal Hemoglobin Expression in Human Adult Erythroblasts through BCL11A Knockdown Using Targeted Endonucleases. Mol Ther Nucleic Acids. 2016 5,e351.

3. Tseng M, Ge S, Roberts R, Kuo C, Choi J, Nissen NN, Kim I, Chu M, Shin B, Toyoda M, Jordan SC. Liver Transplantation in a Patient with CD40 Ligand Deficiency and Hyper IgM Syndrome: Clinical and Immunological Assessments. American Journal of Transplantation. 2016 May;16(5):1626-1632.

4. Hoban MD, Lumaquin D, Kuo CY, Romero Z, Long J, Ho M, Young CS, Mojadidi M, Fitz- Gibbon S, Cooper AR, Lill GR, Urbinati F, Campo-Fernandez B, Bjurstrom CF, Pellegrini M, Hollis RP, Kohn DB.CRISPR/Cas9-mediated correction of the sickle mutation in human hematopoietic stem-progenitor cells. Mol Ther. 2016 Sept; 24(9):1561-1569.

5. de la Morena MT, Leonard D, Torgerson TR, Cabral-Marques O, Slatter M, Aghamohammadi A, Chandra S, Murguia-Favela L, Bonilla FA, Kanariou M, Damrongwatanasuk R, Kuo CY, Dvorak CC, Meyts I, Chen K, Kobrynski L, Kapoor N, Richter D, DiGiovanni D, Dhalla F, Farmaki E, Speckmann C, Español T, Shcherbina A, Hanson IC, Litzman J, Routes JM, Wong M, Fuleihan R, Seneviratne SL, Small TN, Janda A, Bezrodnik L, Seger R, Raccio AG, Edgar JD, Chou J, Abbott JK, van Montfrans J, González-Granado LI, Bunin N, Kutukculer N, Gray P, Seminario G, Pasic S, Aquino V, Wysocki C, Abolhassani H, Dorsey M, Cunningham-Rundles C, Knutsen AP, Sleasman J, Costa Carvalho BT, Condino- Neto A, Grunebaum E, Chapel H, Ochs HD, Filipovich A, Cowan M, Gennery A, Cant A, Notarangelo LD, Roifman CM. Long term outcomes of 176 patients with X-linked hyper IgM syndrome treated with or without hematopoietic cell transplantation. J Allergy Clin Immunol. 2017 Apr;139(4):1282-1292.