Harvard Catalyst Profiles

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Sung-Yun Pai, M.D.

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Research
The research activities and funding listed below are automatically derived from NIH ExPORTER and other sources, which might result in incorrect or missing items. Faculty can login to make corrections and additions.
  1. U01AI126612 (PULSIPHER, MICHAEL A) Aug 24, 2017 - Jul 31, 2024
    NIH/NIAID
    Randomized study of low versus moderate dose busulfan in transplant for severe combined immunodeficiency
    Role: Co-Principal Investigator
  2. U01AI125051 (PAI, SUNG-YUN) Jul 7, 2016 - Jun 30, 2022
    NIH/NIAID
    Gene therapy for SCID-X1 with low dose busulfan and a SIN-lentiviral vector
    Role: Principal Investigator
  3. R34AI108522 (PAI, SUNG-YUN) May 1, 2014 - Apr 30, 2015
    NIH/NIAID
    Dose Finding Study of Busulfan for Newly Diagnosed Infants with SCID
    Role: Principal Investigator
  4. R21AI083907 (PAI, SUNG-YUN) Apr 8, 2009 - Mar 31, 2011
    NIH/NIAID
    Humanized Mouse Models of Severe Combined Immunodeficiency
    Role: Principal Investigator
  5. K08AI050601 (PAI, SUNG-YUN) Apr 1, 2002 - Mar 31, 2007
    NIH/NIAID
    GATA3 in T cell maturation/T helper cell differentiation
    Role: Principal Investigator

Bibliographic
Publications listed below are automatically derived from MEDLINE/PubMed and other sources, which might result in incorrect or missing publications. Faculty can login to make corrections and additions.
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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. Ouahed J, Spencer E, Kotlarz D, Shouval DS, Kowalik M, Peng K, Field M, Grushkin-Lerner L, Pai SY, Bousvaros A, Cho J, Argmann C, Schadt E, Mcgovern DPB, Mokry M, Nieuwenhuis E, Clevers H, Powrie F, Uhlig H, Klein C, Muise A, Dubinsky M, Snapper SB. Very Early Onset Inflammatory Bowel Disease: A Clinical Approach With a Focus on the Role of Genetics and Underlying Immune Deficiencies. Inflamm Bowel Dis. 2020 May 12; 26(6):820-842. PMID: 31833544.
    Citations:    
  2. Burroughs L, Petrovic A, Brazauskas R, Liu X, Griffith LM, Ochs HD, Bleesing J, Edwards S, Dvorak CC, Chaudhury S, Prockop S, Quinones R, Goldman F, Quigg T, Chandrakasan S, Smith AR, Parikh SH, Dávila Saldaña BJ, Thakar MS, Phelan R, Shenoy S, Forbes LR, Martinez CA, Chellapandian D, Shereck E, Miller H, Kapoor N, Barnum JL, Chong H, Shyr D, Chen K, Abu-Arja RF, Shah A, Weinacht K, Moore TB, Joshi A, DeSantes K, Gillio AP, Cuvelier GDE, Keller MD, Rozmus J, Torgerson TR, Pulsipher MA, Haddad E, Sullivan K, Logan BR, Kohn DB, Puck JM, Notarangelo LD, Pai SY, Rawlings D, Cowan MJ. Excellent Outcomes Following Hematopoietic Cell Transplantation for Wiskott-Aldrich Syndrome: A PIDTC Report. Blood. 2020 Apr 08. PMID: 32268350.
    Citations:    
  3. Chan AY, Leiding JW, Liu X, Logan BR, Burroughs LM, Allenspach EJ, Skoda-Smith S, Uzel G, Notarangelo LD, Slatter M, Gennery AR, Smith AR, Pai SY, Jordan MB, Marsh RA, Cowan MJ, Dvorak CC, Craddock JA, Prockop SE, Chandrakasan S, Kapoor N, Buckley RH, Parikh S, Chellapandian D, Oshrine BR, Bednarski JJ, Cooper MA, Shenoy S, Davila Saldana BJ, Forbes LR, Martinez C, Haddad E, Shyr DC, Chen K, Sullivan KE, Heimall J, Wright N, Bhatia M, Cuvelier GDE, Goldman FD, Meyts I, Miller HK, Seidel MG, Vander Lugt MT, Bacchetta R, Weinacht KG, Andolina JR, Caywood E, Chong H, de la Morena MT, Aquino VM, Shereck E, Walter JE, Dorsey MJ, Seroogy CM, Griffith LM, Kohn DB, Puck JM, Pulsipher MA, Torgerson TR. Hematopoietic Cell Transplantation in Patients With Primary Immune Regulatory Disorders (PIRD): A Primary Immune Deficiency Treatment Consortium (PIDTC) Survey. Front Immunol. 2020; 11:239. PMID: 32153572.
    Citations:    
  4. 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. Lentiviral gene therapy for X-linked chronic granulomatous disease. Nat Med. 2020 02; 26(2):200-206. PMID: 31988463.
    Citations:    
  5. Pai SY. Treatment of primary immunodeficiency with allogeneic transplant and gene therapy. Hematology Am Soc Hematol Educ Program. 2019 Dec 06; 2019(1):457-465. PMID: 31808905.
    Citations:    
  6. Verheijen J, Wong SY, Rowe JH, Raymond K, Stoddard J, Delmonte OM, Bosticardo M, Dobbs K, Niemela J, Calzoni E, Pai SY, Choi U, Yamazaki Y, Comeau AM, Janssen E, Henderson L, Hazen M, Berry G, Rosenzweig SD, Aldhekri HH, He M, Notarangelo LD, Morava E. Defining a new immune deficiency syndrome: MAN2B2-CDG. J Allergy Clin Immunol. 2020 Mar; 145(3):1008-1011. PMID: 31775018.
    Citations:    
  7. van der Burg M, Mahlaoui N, Gaspar HB, Pai SY. Universal Newborn Screening for Severe Combined Immunodeficiency (SCID). Front Pediatr. 2019; 7:373. PMID: 31620409.
    Citations:    
  8. Marsh RA, Leiding JW, Logan BR, Griffith LM, Arnold DE, Haddad E, Falcone EL, Yin Z, Patel K, Arbuckle E, Bleesing JJ, Sullivan KE, Heimall J, Burroughs LM, Skoda-Smith S, Chandrakasan S, Yu LC, Oshrine BR, Cuvelier GDE, Thakar MS, Chen K, Teira P, Shenoy S, Phelan R, Forbes LR, Chellapandian D, Dávila Saldaña BJ, Shah AJ, Weinacht KG, Joshi A, Boulad F, Quigg TC, Dvorak CC, Grossman D, Torgerson T, Graham P, Prasad V, Knutsen A, Chong H, Miller H, de la Morena MT, DeSantes K, Cowan MJ, Notarangelo LD, Kohn DB, Stenger E, Pai SY, Routes JM, Puck JM, Kapoor N, Pulsipher MA, Malech HL, Parikh S, Kang EM. Chronic Granulomatous Disease-Associated IBD Resolves and Does Not Adversely Impact Survival Following Allogeneic HCT. J Clin Immunol. 2019 10; 39(7):653-667. PMID: 31376032.
    Citations:    
  9. Farmer JR, Foldvari Z, Ujhazi B, De Ravin SS, Chen K, Bleesing JJH, Schuetz C, Al-Herz W, Abraham RS, Joshi AY, Costa-Carvalho BT, Buchbinder D, Booth C, Reiff A, Ferguson PJ, Aghamohammadi A, Abolhassani H, Puck JM, Adeli M, Cancrini C, Palma P, Bertaina A, Locatelli F, Di Matteo G, Geha RS, Kanariou MG, Lycopoulou L, Tzanoudaki M, Sleasman JW, Parikh S, Pinero G, Fischer BM, Dbaibo G, Unal E, Patiroglu T, Karakukcu M, Al-Saad KK, Dilley MA, Pai SY, Dutmer CM, Gelfand EW, Geier CB, Eibl MM, Wolf HM, Henderson LA, Hazen MM, Bonfim C, Wolska-Kusnierz B, Butte MJ, Hernandez JD, Nicholas SK, Stepensky P, Chandrakasan S, Miano M, Westermann-Clark E, Goda V, Kriván G, Holland SM, Fadugba O, Henrickson SE, Ozen A, Karakoc-Aydiner E, Baris S, Kiykim A, Bredius R, Hoeger B, Boztug K, Pashchenko O, Neven B, Moshous D, Villartay JP, Bousfiha AA, Hill HR, Notarangelo LD, Walter JE. Outcomes and Treatment Strategies for Autoimmunity and Hyperinflammation in Patients with RAG Deficiency. J Allergy Clin Immunol Pract. 2019 Jul - Aug; 7(6):1970-1985.e4. PMID: 30877075.
    Citations:    
  10. Alosaimi MF, Shendi H, Beano A, Stafstrom K, El Hawary R, Meshaal S, Galal N, Pai SY, El-Marsafy A, Geha RS, Chou J. T-cell mitochondrial dysfunction and lymphopenia in DOCK2-deficient patients. J Allergy Clin Immunol. 2019 07; 144(1):306-309.e2. PMID: 30826364.
    Citations:    
  11. Bauer DE, Pai SY. Getting Past HSC Security: Cyclosporine H Gives Lentiviruses an Entry Pass. Cell Stem Cell. 2018 12 06; 23(6):775-776. PMID: 30526877.
    Citations:    Fields:    
  12. Aydin SE, Freeman AF, Al-Herz W, Al-Mousa HA, Arnaout RK, Aydin RC, Barlogis V, Belohradsky BH, Bonfim C, Bredius RG, Chu JI, Ciocarlie OC, Dogu F, Gaspar HB, Geha RS, Gennery AR, Hauck F, Hawwari A, Hickstein DD, Hoenig M, Ikinciogullari A, Klein C, Kumar A, Ifversen MRS, Matthes S, Metin A, Neven B, Pai SY, Parikh SH, Picard C, Renner ED, Sanal Ö, Schulz AS, Schuster F, Shah NN, Shereck EB, Slatter MA, Su HC, van Montfrans J, Woessmann W, Ziegler JB, Albert MH. Hematopoietic Stem Cell Transplantation as Treatment for Patients with DOCK8 Deficiency. J Allergy Clin Immunol Pract. 2019 03; 7(3):848-855. PMID: 30391550.
    Citations:    Fields:    
  13. Clarke EL, Connell AJ, Six E, Kadry NA, Abbas AA, Hwang Y, Everett JK, Hofstaedter CE, Marsh R, Armant M, Kelsen J, Notarangelo LD, Collman RG, Hacein-Bey-Abina S, Kohn DB, Cavazzana M, Fischer A, Williams DA, Pai SY, Bushman FD. T cell dynamics and response of the microbiota after gene therapy to treat X-linked severe combined immunodeficiency. Genome Med. 2018 09 28; 10(1):70. PMID: 30261899.
    Citations:    Fields:    
  14. Dvorak CC, Haddad E, Buckley RH, Cowan MJ, Logan B, Griffith LM, Kohn DB, Pai SY, Notarangelo L, Shearer W, Prockop S, Kapoor N, Heimall J, Chaudhury S, Shyr D, Chandra S, Cuvelier G, Moore T, Shenoy S, Goldman F, Smith AR, Sunkersett G, Vander Lugt M, Caywood E, Quigg T, Torgerson T, Chandrakasan S, Craddock J, Dávila Saldaña BJ, Gillio A, Shereck E, Aquino V, DeSantes K, Knutsen A, Thakar M, Yu L, Puck JM. The genetic landscape of severe combined immunodeficiency in the United States and Canada in the current era (2010-2018). J Allergy Clin Immunol. 2019 01; 143(1):405-407. PMID: 30193840.
    Citations: 1     Fields:    
  15. Haddad E, Logan BR, Griffith LM, Buckley RH, Parrott RE, Prockop SE, Small TN, Chaisson J, Dvorak CC, Murnane M, Kapoor N, Abdel-Azim H, Hanson IC, Martinez C, Bleesing JJH, Chandra S, Smith AR, Cavanaugh ME, Jyonouchi S, Sullivan KE, Burroughs L, Skoda-Smith S, Haight AE, Tumlin AG, Quigg TC, Taylor C, Dávila Saldaña BJ, Keller MD, Seroogy CM, Desantes KB, Petrovic A, Leiding JW, Shyr DC, Decaluwe H, Teira P, Gillio AP, Knutsen AP, Moore TB, Kletzel M, Craddock JA, Aquino V, Davis JH, Yu LC, Cuvelier GDE, Bednarski JJ, Goldman FD, Kang EM, Shereck E, Porteus MH, Connelly JA, Fleisher TA, Malech HL, Shearer WT, Szabolcs P, Thakar MS, Vander Lugt MT, Heimall J, Yin Z, Pulsipher MA, Pai SY, Kohn DB, Puck JM, Cowan MJ, O'Reilly RJ, Notarangelo LD. SCID genotype and 6-month posttransplant CD4 count predict survival and immune recovery. Blood. 2018 10 25; 132(17):1737-1749. PMID: 30154114.
    Citations: 1     Fields:    
  16. Allen CE, Marsh R, Dawson P, Bollard CM, Shenoy S, Roehrs P, Hanna R, Burroughs L, Kean L, Talano JA, Schultz KR, Pai SY, Baker KS, Andolina JR, Stenger EO, Connelly J, Ramirez A, Bryant C, Eapen M, Pulsipher MA. Reduced-intensity conditioning for hematopoietic cell transplant for HLH and primary immune deficiencies. Blood. 2018 09 27; 132(13):1438-1451. PMID: 29997222.
    Citations:    Fields:    
  17. Lee PY, Huang Y, Zhou Q, Schnappauf O, Hershfield MS, Li Y, Ganson NJ, Sampaio Moura N, Delmonte OM, Stone SS, Rivkin MJ, Pai SY, Lyons T, Sundel RP, Hsu VW, Notarangelo LD, Aksentijevich I, Nigrovic PA. Disrupted N-linked glycosylation as a disease mechanism in deficiency of ADA2. J Allergy Clin Immunol. 2018 10; 142(4):1363-1365.e8. PMID: 29936104.
    Citations: 1     Fields:    
  18. Miggelbrink AM, Logan BR, Buckley RH, Parrott RE, Dvorak CC, Kapoor N, Abdel-Azim H, Prockop SE, Shyr D, Decaluwe H, Hanson IC, Gillio A, Dávila Saldaña BJ, Eibel H, Hopkins G, Walter JE, Whangbo JS, Kohn DB, Puck JM, Cowan MJ, Griffith LM, Haddad E, O'Reilly RJ, Notarangelo LD, Pai SY. B-cell differentiation and IL-21 response in IL2RG/JAK3 SCID patients after hematopoietic stem cell transplantation. Blood. 2018 06 28; 131(26):2967-2977. PMID: 29728406.
    Citations:    Fields:    
  19. Biswas A, Shouval DS, Griffith A, Goettel JA, Field M, Kang YH, Konnikova L, Janssen E, Redhu NS, Thrasher AJ, Chatila T, Kuchroo VK, Geha RS, Notarangelo LD, Pai SY, Horwitz BH, Snapper SB. WASP-mediated regulation of anti-inflammatory macrophages is IL-10 dependent and is critical for intestinal homeostasis. Nat Commun. 2018 05 03; 9(1):1779. PMID: 29725003.
    Citations:    Fields:    Translation:HumansAnimalsCells
  20. Rowe JH, Delmonte OM, Keles S, Stadinski BD, Dobbs AK, Henderson LA, Yamazaki Y, Allende LM, Bonilla FA, Gonzalez-Granado LI, Celikbilek Celik S, Guner SN, Kapakli H, Yee C, Pai SY, Huseby ES, Reisli I, Regueiro JR, Notarangelo LD. Patients with CD3G mutations reveal a role for human CD3? in Treg diversity and suppressive function. Blood. 2018 05 24; 131(21):2335-2344. PMID: 29653965.
    Citations:    Fields:    
  21. Barzaghi F, Amaya Hernandez LC, Neven B, Ricci S, Kucuk ZY, Bleesing JJ, Nademi Z, Slatter MA, Ulloa ER, Shcherbina A, Roppelt A, Worth A, Silva J, Aiuti A, Murguia-Favela L, Speckmann C, Carneiro-Sampaio M, Fernandes JF, Baris S, Ozen A, Karakoc-Aydiner E, Kiykim A, Schulz A, Steinmann S, Notarangelo LD, Gambineri E, Lionetti P, Shearer WT, Forbes LR, Martinez C, Moshous D, Blanche S, Fisher A, Ruemmele FM, Tissandier C, Ouachee-Chardin M, Rieux-Laucat F, Cavazzana M, Qasim W, Lucarelli B, Albert MH, Kobayashi I, Alonso L, Diaz De Heredia C, Kanegane H, Lawitschka A, Seo JJ, Gonzalez-Vicent M, Diaz MA, Goyal RK, Sauer MG, Yesilipek A, Kim M, Yilmaz-Demirdag Y, Bhatia M, Khlevner J, Richmond Padilla EJ, Martino S, Montin D, Neth O, Molinos-Quintana A, Valverde-Fernandez J, Broides A, Pinsk V, Ballauf A, Haerynck F, Bordon V, Dhooge C, Garcia-Lloret ML, Bredius RG, Kalwak K, Haddad E, Seidel MG, Duckers G, Pai SY, Dvorak CC, Ehl S, Locatelli F, Goldman F, Gennery AR, Cowan MJ, Roncarolo MG, Bacchetta R. Long-term follow-up of IPEX syndrome patients after different therapeutic strategies: An international multicenter retrospective study. J Allergy Clin Immunol. 2018 03; 141(3):1036-1049.e5. PMID: 29241729.
    Citations: 2     Fields:    
  22. Heimall J, Logan BR, Cowan MJ, Notarangelo LD, Griffith LM, Puck JM, Kohn DB, Pulsipher MA, Parikh S, Martinez C, Kapoor N, O'Reilly R, Boyer M, Pai SY, Goldman F, Burroughs L, Chandra S, Kletzel M, Thakar M, Connelly J, Cuvelier G, Davila Saldana BJ, Shereck E, Knutsen A, Sullivan KE, DeSantes K, Gillio A, Haddad E, Petrovic A, Quigg T, Smith AR, Stenger E, Yin Z, Shearer WT, Fleisher T, Buckley RH, Dvorak CC. Immune reconstitution and survival of 100 SCID patients post-hematopoietic cell transplant: a PIDTC natural history study. Blood. 2017 12 21; 130(25):2718-2727. PMID: 29021228.
    Citations: 6     Fields:    Translation:HumansPHPublic Health
  23. Dobbs K, Tabellini G, Calzoni E, Patrizi O, Martinez P, Giliani SC, Moratto D, Al-Herz W, Cancrini C, Cowan M, Bleesing J, Booth C, Buchbinder D, Burns SO, Chatila TA, Chou J, Daza-Cajigal V, Ott de Bruin LM, de la Morena MT, Di Matteo G, Finocchi A, Geha R, Goyal RK, Hayward A, Holland S, Huang CH, Kanariou MG, King A, Kaplan B, Kleva A, Kuijpers TW, Lee BW, Lougaris V, Massaad M, Meyts I, Morsheimer M, Neven B, Pai SY, Parvaneh N, Plebani A, Prockop S, Reisli I, Soh JY, Somech R, Torgerson TR, Kim YJ, Walter JE, Gennery AR, Keles S, Manis JP, Marcenaro E, Moretta A, Parolini S, Notarangelo LD. Corrigendum: Natural Killer Cells from Patients with Recombinase-Activating Gene and Non-Homologous End Joining Gene Defects Comprise a Higher Frequency of CD56bright NKG2A+++ Cells, and Yet Display Increased Degranulation and Higher Perforin Content. Front Immunol. 2017; 8:1244. PMID: 29042861.
    Citations:    
  24. Dobbs K, Tabellini G, Calzoni E, Patrizi O, Martinez P, Giliani SC, Moratto D, Al-Herz W, Cancrini C, Cowan M, Bleesing J, Booth C, Buchbinder D, Burns SO, Chatila TA, Chou J, Daza-Cajigal V, Ott de Bruin LM, de la Morena M, Di Matteo G, Finocchi A, Geha R, Goyal RK, Hayward A, Holland S, Huang CH, Kanariou MG, King A, Kaplan B, Kleva A, Kuijpers TW, Lee BW, Lougaris V, Massaad M, Meyts I, Morsheimer M, Neven B, Pai SY, Parvaneh N, Plebani A, Prockop S, Reisli I, Soh JY, Somech R, Torgerson TR, Kim YJ, Walter JE, Gennery AR, Keles S, Manis JP, Marcenaro E, Moretta A, Parolini S, Notarangelo LD. Natural Killer Cells from Patients with Recombinase-Activating Gene and Non-Homologous End Joining Gene Defects Comprise a Higher Frequency of CD56bright NKG2A+++ Cells, and Yet Display Increased Degranulation and Higher Perforin Content. Front Immunol. 2017; 8:798. PMID: 28769923.
    Citations: 2     
  25. Ivaturi V, Dvorak CC, Chan D, Liu T, Cowan MJ, Wahlstrom J, Stricherz M, Jennissen C, Orchard PJ, Tolar J, Pai SY, Huang L, Aweeka F, Long-Boyle J. Pharmacokinetics and Model-Based Dosing to Optimize Fludarabine Therapy in Pediatric Hematopoietic Cell Transplant Recipients. Biol Blood Marrow Transplant. 2017 Oct; 23(10):1701-1713. PMID: 28684371.
    Citations:    Fields:    Translation:HumansCTClinical Trials
  26. Hoenig M, Lagresle-Peyrou C, Pannicke U, Notarangelo LD, Porta F, Gennery AR, Slatter M, Cowan MJ, Stepensky P, Al-Mousa H, Al-Zahrani D, Pai SY, Al Herz W, Gaspar HB, Veys P, Oshima K, Imai K, Yabe H, Noroski LM, Wulffraat NM, Sykora KW, Soler-Palacin P, Muramatsu H, Al Hilali M, Moshous D, Debatin KM, Schuetz C, Jacobsen EM, Schulz AS, Schwarz K, Fischer A, Friedrich W, Cavazzana M. Reticular dysgenesis: international survey on clinical presentation, transplantation, and outcome. Blood. 2017 05 25; 129(21):2928-2938. PMID: 28331055.
    Citations: 2     Fields:    Translation:HumansCTClinical Trials
  27. Lee YN, Frugoni F, Dobbs K, Tirosh I, Du L, Ververs FA, Ru H, Ott de Bruin L, Adeli M, Bleesing JH, Buchbinder D, Butte MJ, Cancrini C, Chen K, Choo S, Elfeky RA, Finocchi A, Fuleihan RL, Gennery AR, El-Ghoneimy DH, Henderson LA, Al-Herz W, Hossny E, Nelson RP, Pai SY, Patel NC, Reda SM, Soler-Palacin P, Somech R, Palma P, Wu H, Giliani S, Walter JE, Notarangelo LD. Characterization of T and B cell repertoire diversity in patients with RAG deficiency. Sci Immunol. 2016 12 16; 1(6). PMID: 28783691.
    Citations: 5     Fields:    
  28. Kammermeier J, Lucchini G, Pai SY, Worth A, Rampling D, Amrolia P, Silva J, Chiesa R, Rao K, Noble-Jamieson G, Gasparetto M, Ellershaw D, Uhlig H, Sebire N, Elawad M, Notarangelo L, Shah N, Veys P. Stem cell transplantation for tetratricopeptide repeat domain 7A deficiency: long-term follow-up. Blood. 2016 09 01; 128(9):1306-8. PMID: 27418642.
    Citations: 2     Fields:    Translation:Humans
  29. Al-Herz W, Chu JI, van der Spek J, Raghupathy R, Massaad MJ, Keles S, Biggs CM, Cockerton L, Chou J, Dbaibo G, Elisofon SA, Hanna-Wakim R, Kim HB, Lehmann LE, McDonald DR, Notarangelo LD, Veys P, Chatila TA, Geha RS, Gaspar HB, Pai SY. Hematopoietic stem cell transplantation outcomes for 11 patients with dedicator of cytokinesis 8 deficiency. J Allergy Clin Immunol. 2016 09; 138(3):852-859.e3. PMID: 27130861.
    Citations: 5     Fields:    Translation:HumansCells
  30. Biswas A, Shouval D, Goettel J, Field M, Griffith A, Pai SY, Notarangelo L, Geha R, Snapper S. O-008 Aberrant Anti-inflammatory Macrophage Function and Differentiation in Wiskott-Aldrich Syndrome Protein-Deficient Mice and Humans. Inflamm Bowel Dis. 2016 Mar; 22 Suppl 1:S3. PMID: 26849726.
    Citations:    
  31. Sobh A, Crestani E, Cangemi B, Kane J, Chou J, Pai SY, Notarangelo LD, Al-Herz W, Geha RS, Massaad MJ. Autoimmune lymphoproliferative syndrome caused by a homozygous FasL mutation that disrupts FasL assembly. J Allergy Clin Immunol. 2016 Jan; 137(1):324-327.e2. PMID: 26456038.
    Citations: 2     Fields:    Translation:HumansAnimalsCells
  32. Crestani E, Volpi S, Candotti F, Giliani S, Notarangelo LD, Chu J, Aldave Becerra JC, Buchbinder D, Chou J, Geha RS, Kanariou M, King A, Mazza C, Moratto D, Sokolic R, Garabedian E, Porta F, Putti MC, Wakim RH, Tsitsikov E, Pai SY, Notarangelo LD. Broad spectrum of autoantibodies in patients with Wiskott-Aldrich syndrome and X-linked thrombocytopenia. J Allergy Clin Immunol. 2015 Nov; 136(5):1401-4.e1-3. PMID: 26409660.
    Citations: 6     Fields:    Translation:Humans
  33. Frugoni F, Dobbs K, Felgentreff K, Aldhekri H, Al Saud BK, Arnaout R, Ali AA, Abhyankar A, Alroqi F, Giliani S, Ojeda MM, Tsitsikov E, Pai SY, Casanova JL, Notarangelo LD, Manis JP. A novel mutation in the POLE2 gene causing combined immunodeficiency. J Allergy Clin Immunol. 2016 Feb; 137(2):635-638.e1. PMID: 26365386.
    Citations: 6     Fields:    Translation:HumansCells
  34. Chou J, Badran YR, Yee CSK, Bainter W, Ohsumi TK, Al-Hammadi S, Pai SY, Feske S, Geha RS. A novel mutation in ORAI1 presenting with combined immunodeficiency and residual T-cell function. J Allergy Clin Immunol. 2015 Aug; 136(2):479-482.e1. PMID: 26070885.
    Citations: 5     Fields:    Translation:HumansCells
  35. Bode SF, Ammann S, Al-Herz W, Bataneant M, Dvorak CC, Gehring S, Gennery A, Gilmour KC, Gonzalez-Granado LI, Groß-Wieltsch U, Ifversen M, Lingman-Framme J, Matthes-Martin S, Mesters R, Meyts I, van Montfrans JM, Pachlopnik Schmid J, Pai SY, Soler-Palacin P, Schuermann U, Schuster V, Seidel MG, Speckmann C, Stepensky P, Sykora KW, Tesi B, Vraetz T, Waruiru C, Bryceson YT, Moshous D, Lehmberg K, Jordan MB, Ehl S. The syndrome of hemophagocytic lymphohistiocytosis in primary immunodeficiencies: implications for differential diagnosis and pathogenesis. Haematologica. 2015 Jul; 100(7):978-88. PMID: 26022711.
    Citations: 17     Fields:    Translation:HumansCells
  36. Boisson B, Laplantine E, Dobbs K, Cobat A, Tarantino N, Hazen M, Lidov HG, Hopkins G, Du L, Belkadi A, Chrabieh M, Itan Y, Picard C, Fournet JC, Eibel H, Tsitsikov E, Pai SY, Abel L, Al-Herz W, Casanova JL, Israel A, Notarangelo LD. Human HOIP and LUBAC deficiency underlies autoinflammation, immunodeficiency, amylopectinosis, and lymphangiectasia. J Exp Med. 2015 Jun 01; 212(6):939-51. PMID: 26008899.
    Citations: 38     Fields:    Translation:HumansCells
  37. Goettel JA, Biswas S, Lexmond WS, Yeste A, Passerini L, Patel B, Yang S, Sun J, Ouahed J, Shouval DS, McCann KJ, Horwitz BH, Mathis D, Milford EL, Notarangelo LD, Roncarolo MG, Fiebiger E, Marasco WA, Bacchetta R, Quintana FJ, Pai SY, Klein C, Muise AM, Snapper SB. Fatal autoimmunity in mice reconstituted with human hematopoietic stem cells encoding defective FOXP3. Blood. 2015 Jun 18; 125(25):3886-95. PMID: 25833964.
    Citations: 9     Fields:    Translation:HumansAnimals
  38. Chou J, Massaad MJ, Cangemi B, Bainter W, Platt C, Badran YR, Raphael BP, Kamin DS, Goldsmith JD, Pai SY, Al-Herz W, Geha RS. A novel mutation in ICOS presenting as hypogammaglobulinemia with susceptibility to opportunistic pathogens. J Allergy Clin Immunol. 2015 Sep; 136(3):794-797.e1. PMID: 25678089.
    Citations: 6     Fields:    Translation:HumansCells
  39. Aydin SE, Kilic SS, Aytekin C, Kumar A, Porras O, Kainulainen L, Kostyuchenko L, Genel F, Kütükcüler N, Karaca N, Gonzalez-Granado L, Abbott J, Al-Zahrani D, Rezaei N, Baz Z, Thiel J, Ehl S, Marodi L, Orange JS, Sawalle-Belohradsky J, Keles S, Holland SM, Sanal Ö, Ayvaz DC, Tezcan I, Al-Mousa H, Alsum Z, Hawwari A, Metin A, Matthes-Martin S, Hönig M, Schulz A, Picard C, Barlogis V, Gennery A, Ifversen M, van Montfrans J, Kuijpers T, Bredius R, Dückers G, Al-Herz W, Pai SY, Geha R, Notheis G, Schwarze CP, Tavil B, Azik F, Bienemann K, Grimbacher B, Heinz V, Gaspar HB, Aydin R, Hagl B, Gathmann B, Belohradsky BH, Ochs HD, Chatila T, Renner ED, Su H, Freeman AF, Engelhardt K, Albert MH. DOCK8 deficiency: clinical and immunological phenotype and treatment options - a review of 136 patients. J Clin Immunol. 2015 Feb; 35(2):189-98. PMID: 25627830.
    Citations: 33     Fields:    Translation:HumansCells
  40. Pai SY, Cowan MJ. Stem cell transplantation for primary immunodeficiency diseases: the North American experience. Curr Opin Allergy Clin Immunol. 2014 Dec; 14(6):521-6. PMID: 25259542.
    Citations: 7     Fields:    Translation:Humans
  41. Hacein-Bey-Abina S, Pai SY, Gaspar HB, Armant M, Berry CC, Blanche S, Bleesing J, Blondeau J, de Boer H, Buckland KF, Caccavelli L, Cros G, De Oliveira S, Fernández KS, Guo D, Harris CE, Hopkins G, Lehmann LE, Lim A, London WB, van der Loo JC, Malani N, Male F, Malik P, Marinovic MA, McNicol AM, Moshous D, Neven B, Oleastro M, Picard C, Ritz J, Rivat C, Schambach A, Shaw KL, Sherman EA, Silberstein LE, Six E, Touzot F, Tsytsykova A, Xu-Bayford J, Baum C, Bushman FD, Fischer A, Kohn DB, Filipovich AH, Notarangelo LD, Cavazzana M, Williams DA, Thrasher AJ. A modified ?-retrovirus vector for X-linked severe combined immunodeficiency. N Engl J Med. 2014 Oct 09; 371(15):1407-17. PMID: 25295500.
    Citations: 65     Fields:    Translation:HumansAnimalsCellsCTClinical Trials
  42. Kwan A, Abraham RS, Currier R, Brower A, Andruszewski K, Abbott JK, Baker M, Ballow M, Bartoshesky LE, Bonilla FA, Brokopp C, Brooks E, Caggana M, Celestin J, Church JA, Comeau AM, Connelly JA, Cowan MJ, Cunningham-Rundles C, Dasu T, Dave N, De La Morena MT, Duffner U, Fong CT, Forbes L, Freedenberg D, Gelfand EW, Hale JE, Hanson IC, Hay BN, Hu D, Infante A, Johnson D, Kapoor N, Kay DM, Kohn DB, Lee R, Lehman H, Lin Z, Lorey F, Abdel-Mageed A, Manning A, McGhee S, Moore TB, Naides SJ, Notarangelo LD, Orange JS, Pai SY, Porteus M, Rodriguez R, Romberg N, Routes J, Ruehle M, Rubenstein A, Saavedra-Matiz CA, Scott G, Scott PM, Secord E, Seroogy C, Shearer WT, Siegel S, Silvers SK, Stiehm ER, Sugerman RW, Sullivan JL, Tanksley S, Tierce ML, Verbsky J, Vogel B, Walker R, Walkovich K, Walter JE, Wasserman RL, Watson MS, Weinberg GA, Weiner LB, Wood H, Yates AB, Puck JM, Bonagura VR. Newborn screening for severe combined immunodeficiency in 11 screening programs in the United States. JAMA. 2014 Aug 20; 312(7):729-38. PMID: 25138334.
    Citations: 75     Fields:    Translation:HumansCellsPHPublic Health
  43. Dvorak CC, Hassan A, Slatter MA, Hönig M, Lankester AC, Buckley RH, Pulsipher MA, Davis JH, Güngör T, Gabriel M, Bleesing JH, Bunin N, Sedlacek P, Connelly JA, Crawford DF, Notarangelo LD, Pai SY, Hassid J, Veys P, Gennery AR, Cowan MJ. Comparison of outcomes of hematopoietic stem cell transplantation without chemotherapy conditioning by using matched sibling and unrelated donors for treatment of severe combined immunodeficiency. J Allergy Clin Immunol. 2014 Oct; 134(4):935-943.e15. PMID: 25109802.
    Citations: 19     Fields:    Translation:HumansCells
  44. Pai SY, Logan BR, Griffith LM, Buckley RH, Parrott RE, Dvorak CC, Kapoor N, Hanson IC, Filipovich AH, Jyonouchi S, Sullivan KE, Small TN, Burroughs L, Skoda-Smith S, Haight AE, Grizzle A, Pulsipher MA, Chan KW, Fuleihan RL, Haddad E, Loechelt B, Aquino VM, Gillio A, Davis J, Knutsen A, Smith AR, Moore TB, Schroeder ML, Goldman FD, Connelly JA, Porteus MH, Xiang Q, Shearer WT, Fleisher TA, Kohn DB, Puck JM, Notarangelo LD, Cowan MJ, O'Reilly RJ. Transplantation outcomes for severe combined immunodeficiency, 2000-2009. N Engl J Med. 2014 Jul 31; 371(5):434-46. PMID: 25075835.
    Citations: 82     Fields:    Translation:HumansCells
  45. O'Connell AE, Volpi S, Dobbs K, Fiorini C, Tsitsikov E, de Boer H, Barlan IB, Despotovic JM, Espinosa-Rosales FJ, Hanson IC, Kanariou MG, Martínez-Beckerat R, Mayorga-Sirera A, Mejia-Carvajal C, Radwan N, Weiss AR, Pai SY, Lee YN, Notarangelo LD. Next generation sequencing reveals skewing of the T and B cell receptor repertoires in patients with wiskott-Aldrich syndrome. Front Immunol. 2014; 5:340. PMID: 25101082.
    Citations: 16     
  46. Tai TS, Pai SY, Ho IC. Itm2a, a target gene of GATA-3, plays a minimal role in regulating the development and function of T cells. PLoS One. 2014; 9(5):e96535. PMID: 24831988.
    Citations: 4     Fields:    Translation:AnimalsCells
  47. Pai SY, de Boer H, Massaad MJ, Chatila TA, Keles S, Jabara HH, Janssen E, Lehmann LE, Hanna-Wakim R, Dbaibo G, McDonald DR, Al-Herz W, Geha RS. Flow cytometry diagnosis of dedicator of cytokinesis 8 (DOCK8) deficiency. J Allergy Clin Immunol. 2014 Jul; 134(1):221-3. PMID: 24698323.
    Citations: 13     Fields:    Translation:HumansCells
  48. Lee YN, Frugoni F, Dobbs K, Walter JE, Giliani S, Gennery AR, Al-Herz W, Haddad E, LeDeist F, Bleesing JH, Henderson LA, Pai SY, Nelson RP, El-Ghoneimy DH, El-Feky RA, Reda SM, Hossny E, Soler-Palacin P, Fuleihan RL, Patel NC, Massaad MJ, Geha RS, Puck JM, Palma P, Cancrini C, Chen K, Vihinen M, Alt FW, Notarangelo LD. A systematic analysis of recombination activity and genotype-phenotype correlation in human recombination-activating gene 1 deficiency. J Allergy Clin Immunol. 2014 Apr; 133(4):1099-108. PMID: 24290284.
    Citations: 41     Fields:    Translation:HumansCells
  49. Shearer WT, Dunn E, Notarangelo LD, Dvorak CC, Puck JM, Logan BR, Griffith LM, Kohn DB, O'Reilly RJ, Fleisher TA, Pai SY, Martinez CA, Buckley RH, Cowan MJ. Establishing diagnostic criteria for severe combined immunodeficiency disease (SCID), leaky SCID, and Omenn syndrome: the Primary Immune Deficiency Treatment Consortium experience. J Allergy Clin Immunol. 2014 Apr; 133(4):1092-8. PMID: 24290292.
    Citations: 50     Fields:    Translation:Humans
  50. Griffith LM, Cowan MJ, Notarangelo LD, Kohn DB, Puck JM, Pai SY, Ballard B, Bauer SC, Bleesing JJ, Boyle M, Brower A, Buckley RH, van der Burg M, Burroughs LM, Candotti F, Cant AJ, Chatila T, Cunningham-Rundles C, Dinauer MC, Dvorak CC, Filipovich AH, Fleisher TA, Bobby Gaspar H, Gungor T, Haddad E, Hovermale E, Huang F, Hurley A, Hurley M, Iyengar S, Kang EM, Logan BR, Long-Boyle JR, Malech HL, McGhee SA, Modell F, Modell V, Ochs HD, O'Reilly RJ, Parkman R, Rawlings DJ, Routes JM, Shearer WT, Small TN, Smith H, Sullivan KE, Szabolcs P, Thrasher A, Torgerson TR, Veys P, Weinberg K, Zuniga-Pflucker JC. Primary Immune Deficiency Treatment Consortium (PIDTC) report. J Allergy Clin Immunol. 2014 Feb; 133(2):335-47. PMID: 24139498.
    Citations: 15     Fields:    Translation:HumansPHPublic Health
  51. Savic RM, Cowan MJ, Dvorak CC, Pai SY, Pereira L, Bartelink IH, Boelens JJ, Bredius RG, Wynn RF, Cuvelier GD, Shaw PJ, Slatter MA, Long-Boyle J. Effect of weight and maturation on busulfan clearance in infants and small children undergoing hematopoietic cell transplantation. Biol Blood Marrow Transplant. 2013 Nov; 19(11):1608-14. PMID: 24029650.
    Citations: 13     Fields:    Translation:Humans
  52. Henderson LA, Frugoni F, Hopkins G, de Boer H, Pai SY, Lee YN, Walter JE, Hazen MM, Notarangelo LD. Expanding the spectrum of recombination-activating gene 1 deficiency: a family with early-onset autoimmunity. J Allergy Clin Immunol. 2013 Oct; 132(4):969-71.e1-2. PMID: 23891352.
    Citations: 24     Fields:    Translation:Humans
  53. Chen R, Giliani S, Lanzi G, Mias GI, Lonardi S, Dobbs K, Manis J, Im H, Gallagher JE, Phanstiel DH, Euskirchen G, Lacroute P, Bettinger K, Moratto D, Weinacht K, Montin D, Gallo E, Mangili G, Porta F, Notarangelo LD, Pedretti S, Al-Herz W, Alfahdli W, Comeau AM, Traister RS, Pai SY, Carella G, Facchetti F, Nadeau KC, Snyder M, Notarangelo LD. Whole-exome sequencing identifies tetratricopeptide repeat domain 7A (TTC7A) mutations for combined immunodeficiency with intestinal atresias. J Allergy Clin Immunol. 2013 Sep; 132(3):656-664.e17. PMID: 23830146.
    Citations: 31     Fields:    Translation:HumansAnimals
  54. Dvorak CC, Cowan MJ, Logan BR, Notarangelo LD, Griffith LM, Puck JM, Kohn DB, Shearer WT, O'Reilly RJ, Fleisher TA, Pai SY, Hanson IC, Pulsipher MA, Fuleihan R, Filipovich A, Goldman F, Kapoor N, Small T, Smith A, Chan KW, Cuvelier G, Heimall J, Knutsen A, Loechelt B, Moore T, Buckley RH. The natural history of children with severe combined immunodeficiency: baseline features of the first fifty patients of the primary immune deficiency treatment consortium prospective study 6901. J Clin Immunol. 2013 Oct; 33(7):1156-64. PMID: 23818196.
    Citations: 35     Fields:    Translation:Humans
  55. Leen AM, Bollard CM, Mendizabal AM, Shpall EJ, Szabolcs P, Antin JH, Kapoor N, Pai SY, Rowley SD, Kebriaei P, Dey BR, Grilley BJ, Gee AP, Brenner MK, Rooney CM, Heslop HE. Multicenter study of banked third-party virus-specific T cells to treat severe viral infections after hematopoietic stem cell transplantation. Blood. 2013 Jun 27; 121(26):5113-23. PMID: 23610374.
    Citations: 112     Fields:    Translation:HumansCells
  56. Cattaneo F, Recher M, Masneri S, Baxi SN, Fiorini C, Antonelli F, Wysocki CA, Calderon JG, Eibel H, Smith AR, Bonilla FA, Tsitsikov E, Giliani S, Notarangelo LD, Pai SY. Hypomorphic Janus kinase 3 mutations result in a spectrum of immune defects, including partial maternal T-cell engraftment. J Allergy Clin Immunol. 2013 Apr; 131(4):1136-45. PMID: 23384681.
    Citations: 6     Fields:    Translation:HumansCells
  57. Tai TS, Pai SY, Ho IC. GATA-3 regulates the homeostasis and activation of CD8+ T cells. J Immunol. 2013 Jan 01; 190(1):428-37. PMID: 23225883.
    Citations: 15     Fields:    Translation:AnimalsCells
  58. Kohn DB, Pai SY, Sadelain M. Gene therapy through autologous transplantation of gene-modified hematopoietic stem cells. Biol Blood Marrow Transplant. 2013 Jan; 19(1 Suppl):S64-9. PMID: 23032601.
    Citations: 15     Fields:    Translation:HumansAnimalsCells
  59. Henderson LA, Frugoni F, Hopkins G, Al-Herz W, Weinacht K, Comeau AM, Bonilla FA, Notarangelo LD, Pai SY. First reported case of Omenn syndrome in a patient with reticular dysgenesis. J Allergy Clin Immunol. 2013 Apr; 131(4):1227-30, 1230.e1-3. PMID: 23014587.
    Citations: 7     Fields:    Translation:HumansCells
  60. Recher M, Berglund LJ, Avery DT, Cowan MJ, Gennery AR, Smart J, Peake J, Wong M, Pai SY, Baxi S, Walter JE, Palendira U, Tangye GA, Rice M, Brothers S, Al-Herz W, Oettgen H, Eibel H, Puck JM, Cattaneo F, Ziegler JB, Giliani S, Tangye SG, Notarangelo LD. IL-21 is the primary common ? chain-binding cytokine required for human B-cell differentiation in vivo. Blood. 2011 Dec 22; 118(26):6824-35. PMID: 22039266.
    Citations: 47     Fields:    Translation:HumansCells
  61. Chan K, Davis J, Pai SY, Bonilla FA, Puck JM, Apkon M. A Markov model to analyze cost-effectiveness of screening for severe combined immunodeficiency (SCID). Mol Genet Metab. 2011 Nov; 104(3):383-9. PMID: 21810544.
    Citations: 24     Fields:    Translation:HumansCellsPHPublic Health
  62. Moratto D, Giliani S, Bonfim C, Mazzolari E, Fischer A, Ochs HD, Cant AJ, Thrasher AJ, Cowan MJ, Albert MH, Small T, Pai SY, Haddad E, Lisa A, Hambleton S, Slatter M, Cavazzana-Calvo M, Mahlaoui N, Picard C, Torgerson TR, Burroughs L, Koliski A, Neto JZ, Porta F, Qasim W, Veys P, Kavanau K, Hönig M, Schulz A, Friedrich W, Notarangelo LD. Long-term outcome and lineage-specific chimerism in 194 patients with Wiskott-Aldrich syndrome treated by hematopoietic cell transplantation in the period 1980-2009: an international collaborative study. Blood. 2011 Aug 11; 118(6):1675-84. PMID: 21659547.
    Citations: 48     Fields:    Translation:Humans
  63. de la Fuente MA, Recher M, Rider NL, Strauss KA, Morton DH, Adair M, Bonilla FA, Ochs HD, Gelfand EW, Pessach IM, Walter JE, King A, Giliani S, Pai SY, Notarangelo LD. Reduced thymic output, cell cycle abnormalities, and increased apoptosis of T lymphocytes in patients with cartilage-hair hypoplasia. J Allergy Clin Immunol. 2011 Jul; 128(1):139-146. PMID: 21570718.
    Citations: 8     Fields:    Translation:HumansCells
  64. McDonald DR, Massaad MJ, Johnston A, Keles S, Chatila T, Geha RS, Pai SY. Successful engraftment of donor marrow after allogeneic hematopoietic cell transplantation in autosomal-recessive hyper-IgE syndrome caused by dedicator of cytokinesis 8 deficiency. J Allergy Clin Immunol. 2010 Dec; 126(6):1304-5.e3. PMID: 20810158.
    Citations: 26     Fields:    Translation:HumansCells
  65. Hale JE, Bonilla FA, Pai SY, Gerstel-Thompson JL, Notarangelo LD, Eaton RB, Comeau AM. Identification of an infant with severe combined immunodeficiency by newborn screening. J Allergy Clin Immunol. 2010 Nov; 126(5):1073-4. PMID: 20933257.
    Citations: 22     Fields:    Translation:HumansPHPublic Health
  66. Gerstel-Thompson JL, Wilkey JF, Baptiste JC, Navas JS, Pai SY, Pass KA, Eaton RB, Comeau AM. High-throughput multiplexed T-cell-receptor excision circle quantitative PCR assay with internal controls for detection of severe combined immunodeficiency in population-based newborn screening. Clin Chem. 2010 Sep; 56(9):1466-74. PMID: 20660142.
    Citations: 30     Fields:    Translation:HumansPHPublic Health
  67. Comeau AM, Hale JE, Pai SY, Bonilla FA, Notarangelo LD, Pasternack MS, Meissner HC, Cooper ER, DeMaria A, Sahai I, Eaton RB. Guidelines for implementation of population-based newborn screening for severe combined immunodeficiency. J Inherit Metab Dis. 2010 Oct; 33(Suppl 2):S273-81. PMID: 20490925.
    Citations: 20     Fields:    Translation:HumansPHPublic Health
  68. Pai SY, Notarangelo LD. Hematopoietic cell transplantation for Wiskott-Aldrich syndrome: advances in biology and future directions for treatment. Immunol Allergy Clin North Am. 2010 May; 30(2):179-94. PMID: 20493395.
    Citations: 8     Fields:    Translation:HumansAnimals
  69. Debelenko LV, Perez-Atayde AR, Dubois SG, Grier HE, Pai SY, Shamberger RC, Kozakewich HP. p53+/mdm2- atypical lipomatous tumor/well-differentiated liposarcoma in young children: an early expression of Li-Fraumeni syndrome. Pediatr Dev Pathol. 2010 May-Jun; 13(3):218-24. PMID: 20028212.
    Citations: 3     Fields:    Translation:Humans
  70. Hattori H, Subramanian KK, Sakai J, Jia Y, Li Y, Porter TF, Loison F, Sarraj B, Kasorn A, Jo H, Blanchard C, Zirkle D, McDonald D, Pai SY, Serhan CN, Luo HR. Small-molecule screen identifies reactive oxygen species as key regulators of neutrophil chemotaxis. Proc Natl Acad Sci U S A. 2010 Feb 23; 107(8):3546-51. PMID: 20142487.
    Citations: 52     Fields:    Translation:HumansAnimalsCells
  71. Pai SY, Kim C, Williams DA. Rac GTPases in human diseases. Dis Markers. 2010; 29(3-4):177-87. PMID: 21178276.
    Citations: 18     Fields:    Translation:HumansAnimalsCells
  72. Pei XH, Bai F, Smith MD, Usary J, Fan C, Pai SY, Ho IC, Perou CM, Xiong Y. CDK inhibitor p18(INK4c) is a downstream target of GATA3 and restrains mammary luminal progenitor cell proliferation and tumorigenesis. Cancer Cell. 2009 May 05; 15(5):389-401. PMID: 19411068.
    Citations: 40     Fields:    Translation:HumansAnimalsCells
  73. Sheehan WJ, Delmonte OM, Miller DT, Roberts AE, Bonilla FA, Morra M, Giliani S, Pai SY, Notarangelo LD, Oettgen HC. Novel presentation of Omenn syndrome in association with aniridia. J Allergy Clin Immunol. 2009 Apr; 123(4):966-9. PMID: 19178939.
    Citations: 3     Fields:    Translation:Humans
  74. Ho IC, Tai TS, Pai SY. GATA3 and the T-cell lineage: essential functions before and after T-helper-2-cell differentiation. Nat Rev Immunol. 2009 Feb; 9(2):125-35. PMID: 19151747.
    Citations: 105     Fields:    Translation:HumansAnimalsCells
  75. Permaul P, Narla A, Hornick JL, Pai SY. Allogeneic hematopoietic stem cell transplantation for X-linked ectodermal dysplasia and immunodeficiency: case report and review of outcomes. Immunol Res. 2009; 44(1-3):89-98. PMID: 19225723.
    Citations: 15     Fields:    Translation:Humans
  76. Sung-Yun Pai and Barbara E. Bierer. Cell-Mediated and Humoral Immunity and the Regulation of Immune Responses. Nathan and Oski's Hematology of Infancy and Childhood. 2008.
  77. Pai SY, Levy O, Jabara HH, Glickman JN, Stoler-Barak L, Sachs J, Nurko S, Orange JS, Geha RS. Allogeneic transplantation successfully corrects immune defects, but not susceptibility to colitis, in a patient with nuclear factor-kappaB essential modulator deficiency. J Allergy Clin Immunol. 2008 Dec; 122(6):1113-1118.e1. PMID: 18851875.
    Citations: 18     Fields:    Translation:HumansCells
  78. Kouros-Mehr H, Bechis SK, Slorach EM, Littlepage LE, Egeblad M, Ewald AJ, Pai SY, Ho IC, Werb Z. GATA-3 links tumor differentiation and dissemination in a luminal breast cancer model. Cancer Cell. 2008 Feb; 13(2):141-52. PMID: 18242514.
    Citations: 147     Fields:    Translation:AnimalsCells
  79. Pai SY, Kang BY, Sabadini AM, Parisini E, Truitt ML, Ho IC. Distinct structural requirements of GATA-3 for the regulation of thymocyte and Th2 cell differentiation. J Immunol. 2008 Jan 15; 180(2):1050-9. PMID: 18178845.
    Citations: 6     Fields:    Translation:AnimalsCells
  80. Pai SY, Kang BY, Sabadini A , Parisini E, Truitt ML, Ho IC. Distinct Structural Requirements of GATA-3 for the Regulation of Thymocyte and Th2 Cell Differentiation. Journal of Immunology. 2008; 180:1050-1059.
  81. Pai SY, Levy O, Jabara HH, Glickman JN, Sachs J, Nurko S, Orange JS, Geha RS. Successful correction of immune defects in a patient with NEMO mutation by allogeneic transplantation uncovers intrinsic epithelial basis for colitis. Journal of Allergy and Clinical Immunology. 2008.
  82. Jennifer M. Puck, SCID Newborn Screening Working Group. Population-based newborn screening for severe combined immunodeficiency: steps toward implementation. J Allergy Clin Immunol. 2007; 120(4):760-8.
  83. Ho IC, Pai SY. GATA-3 - not just for Th2 cells anymore. Cell Mol Immunol. 2007 Feb; 4(1):15-29. PMID: 17349208.
    Citations: 31     Fields:    Translation:HumansAnimalsCells
  84. de Guzman Strong C, Wertz PW, Wang C, Yang F, Meltzer PS, Andl T, Millar SE, Ho IC, Pai SY, Segre JA. Lipid defect underlies selective skin barrier impairment of an epidermal-specific deletion of Gata-3. J Cell Biol. 2006 Nov 20; 175(4):661-70. PMID: 17116754.
    Citations: 31     Fields:    Translation:AnimalsCells
  85. Strong CD, Wertz PW, Wang C, Yang F, Meltzer PS, Andl T, Millar SE, Ho IC, Pai SY, Segre JA. Lipid Defect Underlies Selective Barrier Deficiency in an Epidermal-specific Deletion of Gata-3. J Cell Biol. 2006; 175(4):661-670.
  86. Kim PJ, Pai SY, Brigl M, Besra GS, Gumperz J, Ho IC. GATA-3 regulates the development and function of invariant NKT cells. J Immunol. 2006 Nov 15; 177(10):6650-9. PMID: 17082577.
    Citations: 51     Fields:    Translation:AnimalsCells
  87. Pai SY, DeMartiis D, Forino C, Cavagnini S, Lanfranchi A, Giliani S, Moratto D, Mazza C, Porta F, Imberti L, Notarangelo LD, Mazzolari E. Stem cell transplantation for the Wiskott-Aldrich syndrome: a single-center experience confirms efficacy of matched unrelated donor transplantation. Bone Marrow Transplant. 2006 Nov; 38(10):671-9. PMID: 17013426.
    Citations: 19     Fields:    Translation:Humans
  88. Pai SY, Truitt ML, Ho IC. GATA-3 deficiency abrogates the development and maintenance of T helper type 2 cells. Proc Natl Acad Sci U S A. 2004 Feb 17; 101(7):1993-8. PMID: 14769923.
    Citations: 108     Fields:    Translation:AnimalsCells
  89. Pai SY, Truitt ML, Ting CN, Leiden JM, Glimcher LH, Ho IC. GATA-3 has multiple roles in thymocyte development. Keystone Symposia: T Cell Development for Oral Presentation. 2004.
  90. Pai SY, Truitt ML, Ting CN, Leiden JM, Glimcher LH, Ho IC. Critical roles for transcription factor GATA-3 in thymocyte development. Immunity. 2003 Dec; 19(6):863-75. PMID: 14670303.
    Citations: 111     Fields:    Translation:AnimalsCells
  91. Pai SY, Ho IC. c-Rel delivers a one-two punch in Th1 cell differentiation. J Clin Invest. 2002 Sep; 110(6):741-2. PMID: 12235102.
    Citations: 1     Fields:    Translation:HumansAnimalsCells
  92. Fruman DA, Pai SY, Klee CB, Burakoff SJ, Bierer BE. Measurement of Calcineurin Phosphatase Activity in Cell Extracts Methods. 1996 Apr; 9(2):146-54. PMID: 8812656.
    Citations: 15     Fields:    
  93. Fruman DA, Pai SY, Burakoff SJ, Bierer BE. Characterization of a mutant calcineurin A alpha gene expressed by EL4 lymphoma cells. Mol Cell Biol. 1995 Jul; 15(7):3857-63. PMID: 7791792.
    Citations: 9     Fields:    Translation:AnimalsCells
  94. Pai SY, Fruman DA, Leong T, Neuberg D, Rosano TG, McGarigle C, Antin JH, Bierer BE. Inhibition of calcineurin phosphatase activity in adult bone marrow transplant patients treated with cyclosporine A. Blood. 1994 Dec 01; 84(11):3974-9. PMID: 7949153.
    Citations: 5     Fields:    Translation:HumansCells
  95. Pai SY, Calvo V, Wood M, Bierer BE. Cross-linking CD28 leads to activation of 70-kDa S6 kinase. Eur J Immunol. 1994 Oct; 24(10):2364-8. PMID: 7523138.
    Citations: 4     Fields:    Translation:HumansCells
  96. Pai SY, Calvo V, Wood M, Bierer BE. Cross-linking CD28 leads to activation of 70-kDa S6 kinase. Oral Presentation, American Society of Hematology. 1993; 82(11).
  97. Pai SY, Fruman DA, Leong T, Neuberg D, Rosano TG, McGarigle C, Antin JH, Bierer BE. Inhibition of calcineurin phosphatase activity in adult bone marrow transplant patients treated with cyclosporine A. Oral Presentation, American Society of Hematology. 1993; 82(11).
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Funded by the NIH National Center for Advancing Translational Sciences through its Clinical and Translational Science Awards Program, grant number UL1TR002541.