Caroline Kuo
Dr. Caroline Kuo holds the Pennington Family Foundation Endowed Term Chair in Pediatrics.
Publications:
Luthers CR, Ha SM, Mittelhauser A, Morselli M, Long JD, Kuo CY, Romero Z, Kohn DB. DNA contamination within recombinant adeno-associated virus preparations correlates with decreased CD34+ cell clonogenic potential. Mol Ther Methods Clin Dev. 2024 Dec 12; 32(4):101334. view on PubMed
Rai E, Doroudchi A, Nulsen B, Younes R, Kuo CY. Effective Management of XLA Associated Enteropathy with Vedolizumab Monotherapy. J Clin Immunol. 2024 Jun 19; 44(7):150. view on PubMed
Mudde ACA, Kuo CY, Kohn DB, Booth C. What a Clinician Needs to Know About Genome Editing: Status and Opportunities for Inborn Errors of Immunity. J Allergy Clin Immunol Pract. 2024 May; 12(5):1139-1149. view on PubMed
Cowan MJ, Yu J, Facchino J, Fraser-Browne C, Sanford U, Kawahara M, Dara J, Long-Boyle J, Oh J, Chan W, Chag S, Broderick L, Chellapandian D, Decaluwe H, Golski C, Hu D, Kuo CY, Miller HK, Petrovic A, Currier R, Hilton JF, Punwani D, Dvorak CC, Malech HL, McIvor RS, Puck JM. Lentiviral Gene Therapy for Artemis-Deficient SCID. N Engl J Med. 2022 12 22; 387(25):2344-2355. view on PubMed
Vogel B, Boyd NK, Kuo CY, Kohn LA, Soldatos A, Santoro JD. Immunotherapy responsive neuroinflammation in a child with FAS-associated death-domain mutation. Clin Genet. 2023 02; 103(2):252-253. view on PubMed
Cuvelier GDE, Logan BR, Prockop SE, Buckley RH, Kuo CY, Griffith LM, Liu X, Yip A, Hershfield MS, Ayoub PG, Moore TB, Dorsey MJ, O'Reilly RJ, Kapoor N, Pai SY, Kapadia M, Ebens CL, Forbes Satter LR, Burroughs LM, Petrovic A, Chellapandian D, Heimall J, Shyr DC, Rayes A, Bednarski JJ, Chandra S, Chandrakasan S, Gillio AP, Madden L, Quigg TC, Caywood EH, Dávila Saldaña BJ, DeSantes K, Eissa H, Goldman FD, Rozmus J, Shah AJ, Vander Lugt MT, Thakar MS, Parrott RE, Martinez C, Leiding JW, Torgerson TR, Pulsipher MA, Notarangelo LD, Cowan MJ, Dvorak CC, Haddad E, Puck JM, Kohn DB. Outcomes following treatment for ADA-deficient severe combined immunodeficiency: a report from the PIDTC. Blood. 2022 08 18; 140(7):685-705. view on PubMed
Long JD, Trope EC, Yang J, Rector K, Kuo CY. Genes as Medicine: The Development of Gene Therapies for Inborn Errors of Immunity. Hematol Oncol Clin North Am. 2022 08; 36(4):829-851. view on PubMed
Meer E, Solanes F, Kohn L, Kuo CY, Wong DA, Pineles S, Tsui I. Ocular findings associated with FADD deficiency resemble familial exudative vitreoretinopathy. Am J Ophthalmol Case Rep. 2022 Mar; 25:101305. view on PubMed
Gray DH, Santos J, Keir AG, Villegas I, Maddock S, Trope EC, Long JD, Kuo CY. A comparison of DNA repair pathways to achieve a site-specific gene modification of the Bruton's tyrosine kinase gene. Mol Ther Nucleic Acids. 2022 Mar 08; 27:505-516. view on PubMed
Kuo CY. Hematopoietic stem cell transplant for the treatment of adenosine deaminase-deficient severe combined immune deficiency. Ann Allergy Asthma Immunol. 2021 05; 126(5):449-450. view on PubMed
Gray DH, Villegas I, Long J, Santos J, Keir A, Abele A, Kuo CY, Kohn DB. Optimizing Integration and Expression of Transgenic Bruton's Tyrosine Kinase for CRISPR-Cas9-Mediated Gene Editing of X-Linked Agammaglobulinemia. CRISPR J. 2021 04; 4(2):191-206. view on PubMed
Kuo CY, Garabedian E, Puck J, Cowan MJ, Sullivan KE, Buckley RH, Cunningham-Rundles C, Marsh R, Candotti F, Kohn DB. Adenosine Deaminase (ADA)-Deficient Severe Combined Immune Deficiency (SCID) in the US Immunodeficiency Network (USIDNet) Registry. J Clin Immunol. 2020 11; 40(8):1124-1131. view on PubMed
Kuo CY, Kohn DB. Overview of the current status of gene therapy for primary immune deficiencies (PIDs). J Allergy Clin Immunol. 2020 08; 146(2):229-233. view on PubMed
Kohn LA, Long JD, Trope EC, Kuo CY. Novel Compound Heterozygote Variations in FADD Identified to Cause FAS-Associated Protein with Death Domain Deficiency. J Clin Immunol. 2020 05; 40(4):658-661. view on PubMed
Kohn DB, Booth C, Kang EM, Pai SY, Shaw KL, Santilli G, Armant M, Buckland KF, Choi U, De Ravin SS, Dorsey MJ, Kuo CY, Leon-Rico D, Rivat C, Izotova N, Gilmour K, Snell K, Dip JX, Darwish J, Morris EC, Terrazas D, Wang LD, Bauser CA, Paprotka T, Kuhns DB, Gregg J, Raymond HE, Everett JK, Honnet G, Biasco L, Newburger PE, Bushman FD, Grez M, Gaspar HB, Williams DA, Malech HL, Galy A, Thrasher AJ, Net4CGD consortium. Lentiviral gene therapy for X-linked chronic granulomatous disease. Nat Med. 2020 02; 26(2):200-206. view on PubMed
Romero Z, Lomova A, Said S, Miggelbrink A, Kuo CY, Campo-Fernandez B, Hoban MD, Masiuk KE, Clark DN, Long J, Sanchez JM, Velez M, Miyahira E, Zhang R, Brown D, Wang X, Kurmangaliyev YZ, Hollis RP, Kohn DB. Editing the Sickle Cell Disease Mutation in Human Hematopoietic Stem Cells: Comparison of Endonucleases and Homologous Donor Templates. Mol Ther. 2019 08 07; 27(8):1389-1406. view on PubMed
Kuo CY. Advances in site-specific gene editing for primary immune deficiencies. Curr Opin Allergy Clin Immunol. 2018 12; 18(6):453-458. view on PubMed
Kuo CY, Signer R, Saitta SC. Immune and Genetic Features of the Chromosome 22q11.2 Deletion (DiGeorge Syndrome). Curr Allergy Asthma Rep. 2018 10 30; 18(12):75. view on PubMed
Csukasi F, Duran I, Barad M, Barta T, Gudernova I, Trantirek L, Martin JH, Kuo CY, Woods J, Lee H, Cohn DH, Krejci P, Krakow D. The PTH/PTHrP-SIK3 pathway affects skeletogenesis through altered mTOR signaling. Sci Transl Med. 2018 09 19; 10(459). view on PubMed
Belderbos ME, Gennery AR, Dvorak CC, Blok HJ, Eikema DJ, Silva JMF, Veys P, Neven B, Buckley R, Cole T, Cowan MJ, Goebel WS, Hoenig M, Kuo CY, Stiehm ER, Wynn R, Bierings M, Inborn Errors Working Party of the European Group for Blood and Marrow Transplantation and the Prima. Outcome of domino hematopoietic stem cell transplantation in human subjects: An international case series. J Allergy Clin Immunol. 2018 11; 142(5):1628-1631.e4. view on PubMed
Kuo CY, Long JD, Campo-Fernandez B, de Oliveira S, Cooper AR, Romero Z, Hoban MD, Joglekar AV, Lill GR, Kaufman ML, Fitz-Gibbon S, Wang X, Hollis RP, Kohn DB. Site-Specific Gene Editing of Human Hematopoietic Stem Cells for X-Linked Hyper-IgM Syndrome. Cell Rep. 2018 05 29; 23(9):2606-2616. view on PubMed
Long J, Hoban MD, Cooper AR, Kaufman ML, Kuo CY, Campo-Fernandez B, Lumaquin D, Hollis RP, Wang X, Kohn DB, Romero Z. Characterization of Gene Alterations following Editing of the β-Globin Gene Locus in Hematopoietic Stem/Progenitor Cells. Mol Ther. 2018 02 07; 26(2):468-479. view on PubMed
Kohn DB, Kuo CY. New frontiers in the therapy of primary immunodeficiency: From gene addition to gene editing. J Allergy Clin Immunol. 2017 Mar; 139(3):726-732. view on PubMed
Kuo CY, Garcia-Lloret MI, Slev P, Bohnsack JF, Chen K. Profound T-cell lymphopenia associated with prenatal exposure to purine antagonists detected by TREC newborn screening. J Allergy Clin Immunol Pract. 2017 Jan - Feb; 5(1):198-200. view on PubMed
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. view on PubMed
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 CD34+ cells. Mol Ther. 2016 Sep; 24(9):1561-9. view on PubMed
Kuo CY, Kohn DB. Gene Therapy for the Treatment of Primary Immune Deficiencies. Curr Allergy Asthma Rep. 2016 May; 16(5):39. view on PubMed
Kuo CY, Chase J, Garcia Lloret M, Stiehm ER, Moore T, Aguilera MJ, Lopez Siles J, Church JA. Newborn screening for severe combined immunodeficiency does not identify bare lymphocyte syndrome. J Allergy Clin Immunol. 2013 Jun; 131(6):1693-5. view on PubMed
