Caroline Yi-Shoo Kuo

Title(s)Assistant Professor-in-Residence, Pediatrics
SchoolMedicine
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    Dr. Caroline Kuo holds the Pennington Family Foundation Endowed Term Chair in Pediatrics.

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    Publications listed below are automatically derived from MEDLINE/PubMed and other sources, which might result in incorrect or missing publications. Researchers can login to make corrections and additions, or contact us for help. to make corrections and additions.
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    Altmetrics Details PMC Citations indicate the number of times the publication was cited by articles in PubMed Central, and the Altmetric score represents citations in news articles and social media. (Note that publications are often cited in additional ways that are not shown here.) Fields are based on how the National Library of Medicine (NLM) classifies the publication's journal and might not represent the specific topic of the publication. Translation tags are based on the publication type and the MeSH terms NLM assigns to the publication. Some publications (especially newer ones and publications not in PubMed) might not yet be assigned Field or Translation tags.) Click a Field or Translation tag to filter the publications.
    1. Congenital Disorder of Glycosylation in a 40-Year-Old Male with Hypogammaglobulinemia. Clin Chem. 2025 Jul 04; 71(7):821-823. Wazeerud-Din IJ, Wong DA, Zhang W, Kuo CY, He M. PMID: 40613798; PMCID: PMC12231354.
      View in: PubMed   Mentions:    Fields:    
    2. CD40 ligand: the essential link in B/T cell collaboration and immune activation. J Immunol. 2025 Jul 01; 214(7):1441-1443. Kuo CY. PMID: 40262278.
      View in: PubMed   Mentions:    Fields:    Translation:HumansAnimalsCells
    3. Clinical and Molecular Characteristics of X-Linked Agammaglobulinemia Patients 55 Years or Older. J Allergy Clin Immunol Pract. 2025 Jun 24. Chin AT, Ochs HD, Kobayashi R, Abolhassani H, Alachkar H, Barmettler S, Baxendale H, Boiling K, Catanzaro J, Chua I, Coulter T, Cunningham-Rundles C, Elcombe SE, Fischer A, Grimbacher B, Gupta S, Herriot R, Herwadkar A, Imai K, Inoue S, Kirkpatrick C, Knutsen AP, Kumararatne D, Lea E, Lin MW, Litzman J, Mahlaoui N, Moriya K, Nonoyama S, Patel S, Perez E, Quinti I, Hostoffer RW, Rothenfusser S, Sargur R, Shields A, Sogkas G, Suan D, Tan T, Thomas M, Warnatz K, Younger EM, Kuo CY. PMID: 40571245.
      View in: PubMed   Mentions:    Fields:    
    4. Lentiviral Gene Therapy for Severe Leukocyte Adhesion Deficiency Type 1. N Engl J Med. 2025 May 01; 392(17):1698-1709. Booth C, Sevilla J, Almarza E, Kuo CY, Zubicaray J, Terrazas D, O'Toole G, Chitty-Lopez M, Choi G, Nicoletti E, Long-Boyle J, Fernandes A, Chetty K, De Oliveira S, Banuelos C, Xu-Bayford J, Bastone AL, John-Neek P, Jackson C, Moore TB, Gilmour K, Schambach A, Rothe M, Kasbekar S, Rao GR, Patel K, Shah G, Thrasher AJ, Bueren JA, Schwartz JD, Kohn DB. PMID: 40305711.
      View in: PubMed   Mentions:    Fields:    Translation:HumansCellsCTClinical Trials
    5. 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. Luthers CR, Ha SM, Mittelhauser A, Morselli M, Long JD, Kuo CY, Romero Z, Kohn DB. PMID: 39381161; PMCID: PMC11460252.
      View in: PubMed   Mentions: 1  
    6. Effective Management of XLA Associated Enteropathy with Vedolizumab Monotherapy. J Clin Immunol. 2024 06 19; 44(7):150. Rai E, Doroudchi A, Nulsen B, Younes R, Kuo CY. PMID: 38896327.
      View in: PubMed   Mentions:    Fields:    Translation:Humans
    7. 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. Mudde ACA, Kuo CY, Kohn DB, Booth C. PMID: 38246560.
      View in: PubMed   Mentions:    Fields:    Translation:HumansAnimals
    8. Lentiviral Gene Therapy for Artemis-Deficient SCID. N Engl J Med. 2022 12 22; 387(25):2344-2355. 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. PMID: 36546626; PMCID: PMC9884487.
      View in: PubMed   Mentions: 22     Fields:    Translation:HumansCellsCTClinical Trials
    9. Immunotherapy responsive neuroinflammation in a child with FAS-associated death-domain mutation. Clin Genet. 2023 02; 103(2):252-253. Vogel B, Boyd NK, Kuo CY, Kohn LA, Soldatos A, Santoro JD. PMID: 36254101.
      View in: PubMed   Mentions:    Fields:    Translation:HumansCells
    10. Outcomes following treatment for ADA-deficient severe combined immunodeficiency: a report from the PIDTC. Blood. 2022 08 18; 140(7):685-705. 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. PMID: 35671392; PMCID: PMC9389638.
      View in: PubMed   Mentions: 15     Fields:    Translation:HumansCTClinical Trials
    11. Genes as Medicine: The Development of Gene Therapies for Inborn Errors of Immunity. Hematol Oncol Clin North Am. 2022 08; 36(4):829-851. Long JD, Trope EC, Yang J, Rector K, Kuo CY. PMID: 35778331.
      View in: PubMed   Mentions: 1     Fields:    Translation:Humans
    12. Ocular findings associated with FADD deficiency resemble familial exudative vitreoretinopathy. Am J Ophthalmol Case Rep. 2022 Mar; 25:101305. Meer E, Solanes F, Kohn L, Kuo CY, Wong DA, Pineles S, Tsui I. PMID: 35243129; PMCID: PMC8859737.
      View in: PubMed   Mentions: 3  
    13. 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. Gray DH, Santos J, Keir AG, Villegas I, Maddock S, Trope EC, Long JD, Kuo CY. PMID: 35036061; PMCID: PMC8728535.
      View in: PubMed   Mentions: 5  
    14. 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. Kuo CY. PMID: 33941315.
      View in: PubMed   Mentions:    Fields:    Translation:Humans
    15. 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. Gray DH, Villegas I, Long J, Santos J, Keir A, Abele A, Kuo CY, Kohn DB. PMID: 33876953; PMCID: PMC8336228.
      View in: PubMed   Mentions: 14     Fields:    Translation:HumansCells
    16. 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. Kuo CY, Garabedian E, Puck J, Cowan MJ, Sullivan KE, Buckley RH, Cunningham-Rundles C, Marsh R, Candotti F, Kohn DB. PMID: 32880085; PMCID: PMC8216639.
      View in: PubMed   Mentions: 14     Fields:    Translation:HumansPHPublic Health
    17. Overview of the current status of gene therapy for primary immune deficiencies (PIDs). J Allergy Clin Immunol. 2020 08; 146(2):229-233. Kuo CY, Kohn DB. PMID: 32771134.
      View in: PubMed   Mentions: 5     Fields:    Translation:HumansAnimals
    18. 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. Kohn LA, Long JD, Trope EC, Kuo CY. PMID: 32350755; PMCID: PMC7253512.
      View in: PubMed   Mentions: 8     Fields:    Translation:HumansCells
    19. Lentiviral gene therapy for X-linked chronic granulomatous disease. Nat Med. 2020 02; 26(2):200-206. 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. PMID: 31988463; PMCID: PMC7115833.
      View in: PubMed   Mentions: 116     Fields:    Translation:HumansCellsCTClinical Trials
    20. 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. 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. PMID: 31178391; PMCID: PMC6697408.
      View in: PubMed   Mentions: 74     Fields:    Translation:HumansCells
    21. Advances in site-specific gene editing for primary immune deficiencies. Curr Opin Allergy Clin Immunol. 2018 12; 18(6):453-458. Kuo CY. PMID: 30299399.
      View in: PubMed   Mentions: 4     Fields:    Translation:HumansAnimals
    22. Immune and Genetic Features of the Chromosome 22q11.2 Deletion (DiGeorge Syndrome). Curr Allergy Asthma Rep. 2018 10 30; 18(12):75. Kuo CY, Signer R, Saitta SC. PMID: 30377837.
      View in: PubMed   Mentions: 11     Fields:    Translation:HumansCells
    23. The PTH/PTHrP-SIK3 pathway affects skeletogenesis through altered mTOR signaling. Sci Transl Med. 2018 09 19; 10(459). 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. PMID: 30232230; PMCID: PMC7529349.
      View in: PubMed   Mentions: 23     Fields:    Translation:HumansCells
    24. 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. 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. PMID: 29981805; PMCID: PMC6226332.
      View in: PubMed   Mentions:    Fields:    Translation:Humans
    25. Site-Specific Gene Editing of Human Hematopoietic Stem Cells for X-Linked Hyper-IgM Syndrome. Cell Rep. 2018 05 29; 23(9):2606-2616. 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. PMID: 29847792; PMCID: PMC6181643.
      View in: PubMed   Mentions: 85     Fields:    Translation:HumansAnimalsCells
    26. 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. Long J, Hoban MD, Cooper AR, Kaufman ML, Kuo CY, Campo-Fernandez B, Lumaquin D, Hollis RP, Wang X, Kohn DB, Romero Z. PMID: 29221806; PMCID: PMC5835144.
      View in: PubMed   Mentions: 17     Fields:    Translation:HumansCells
    27. New frontiers in the therapy of primary immunodeficiency: From gene addition to gene editing. J Allergy Clin Immunol. 2017 Mar; 139(3):726-732. Kohn DB, Kuo CY. PMID: 28270364; PMCID: PMC5911283.
      View in: PubMed   Mentions: 22     Fields:    Translation:HumansAnimals
    28. 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. Kuo CY, Garcia-Lloret MI, Slev P, Bohnsack JF, Chen K. PMID: 28065337; PMCID: PMC5930011.
      View in: PubMed   Mentions: 9     Fields:    Translation:HumansCellsPHPublic Health
    29. 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. 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. PMID: 27697500; PMCID: PMC5374029.
      View in: PubMed   Mentions: 53     Fields:    Translation:Humans
    30. CRISPR/Cas9-Mediated Correction of the Sickle Mutation in Human CD34+ cells. Mol Ther. 2016 Sep; 24(9):1561-9. 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. PMID: 27406980; PMCID: PMC5113113.
      View in: PubMed   Mentions: 94     Fields:    Translation:HumansCells
    31. Gene Therapy for the Treatment of Primary Immune Deficiencies. Curr Allergy Asthma Rep. 2016 May; 16(5):39. Kuo CY, Kohn DB. PMID: 27056559; PMCID: PMC5909980.
      View in: PubMed   Mentions: 23     Fields:    Translation:HumansAnimalsCells
    32. Newborn screening for severe combined immunodeficiency does not identify bare lymphocyte syndrome. J Allergy Clin Immunol. 2013 Jun; 131(6):1693-5. Kuo CY, Chase J, Garcia Lloret M, Stiehm ER, Moore T, Aguilera MJ, Lopez Siles J, Church JA. PMID: 23453137.
      View in: PubMed   Mentions: 16     Fields:    Translation:HumansPHPublic Health
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