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Prof Bobby Gaspar

  • Telephone:

    0044 207 905 2319

  • Extension:

    42319

  • Fax:

    0044 207 905 2810

  • Email:

    h.gaspar@ucl.ac.uk

  • Address:

    30,
    Guilford Street,
    London ,
    WC1N 1EH

  • Appointments:

    Professor of Paediatrics and Immunology, ICH - Molecular Immunology Unit, Dept of Infection & Immunity

Research Summary

Research in my group spans many aspects of primary immunodeficiencies including genetic diagnosis, molecular and cellular pathogenesis, haematopoietic stem cell transplantation and development of gene therapy. Current lab projects: 1) Understanding the molecular basis of TACI defects in Common Variable Immunodeficiency using in vitro and in vivo models 2) Development of T cell strategies to improve outcome following stem cell transplantation including genetic modification of T cells using suicide genes and generation of virus specific T cells 3) Development of gene therapy models for X-linked lymphoproliferative disease (XLP) and haemophagocytic lymphohistiocytosis (HLH) 4) Gene therapy for severe combined immunodeficiency (X-linked SCID and ADA SCID) + this includes implementation of clinical trials and development of new safer viral constructs Clinical projects 1) Outcome of stem cell transplantation for ADA deficiency 2) Understanding the systemic nature of ADA deficiency 3) Long term cognitive outcome in patients receiving stem cell transplants

Research Activities

  • Pathogenesis and management of primary immunodefic
  • T cell therapies

Recent Publications

Displaying 50 most recent publications. For the full list please visit UCL Discovery

  1. Gaspar HB,Swift S,Thrasher AJ (2013) "Special exemptions": should they be put on trial? Mol Ther, 21(2), 261 - 262. 10.1038/mt.2013.1.
  2. Qasim W,Gilmour K,Zhan H,Derniame S,McNicol AM,Ip W,Hiwarkar P,Veys P,Gaspar HB (2013) Interferon-γ capture T cell therapy for persistent Adenoviraemia following allogeneic haematopoietic stem cell transplantation. Br J Haematol, 161(3), 449 - 452. 10.1111/bjh.12251.
  3. Rivat C,Booth C,Alonso-Ferrero M,Blundell M,Sebire NJ,Thrasher AJ,Gaspar HB (2013) SAP gene transfer restores cellular and humoral immune function in a murine model of X-linked lymphoproliferative disease. Blood, 121(7), 1073 - 1076. 10.1182/blood-2012-07-445858.
  4. Montiel-Equihua CA,Thrasher AJ,Gaspar HB (2012) Gene therapy for severe combined immunodeficiency due to adenosine deaminase deficiency. Curr Gene Ther, 12(1), 57 - 65.
  5. Montiel-Equihua CA,Zhang L,Knight S,Saadeh H,Scholz S,Carmo M,Alonso-Ferrero ME,Blundell MP,Monkeviciute A,Schulz R,Collins M,Takeuchi Y,Schmidt M,Fairbanks L,Antoniou M,Thrasher AJ,Gaspar HB (2012) The β-globin locus control region in combination with the EF1α short promoter allows enhanced lentiviral vector-mediated erythroid gene expression with conserved multilineage activity. Mol Ther, 20(7), 1400 - 1409. 10.1038/mt.2012.50.
  6. Rivat C,Booth C,Blundell M,Thrasher AJ,Gaspar HB (2012) Lentiviral Vector Mediated Gene Therapy for X-Linked Lymphoproliferative Disease Restores Humoral and Cellular Functions MOLECULAR THERAPY, 20, S87 - S88.
  7. Montiel-Equihua CA,Zhang L,Knight S,Saadeh H,Scholz S,Carmo M,Alonso-Ferrero ME,Blundell MP,Monkeviciute A,Schulz R,Collins M,Takeuchi Y,Schmidt M,Fairbanks L,Antoniou M,Thrasher AJ,Gaspar HB (2012) Development of a gene expression system for enhanced erythroid expression HUMAN GENE THERAPY, 23(5), A11 - A11.
  8. Rivat C,Booth C,Blundell M,Thrasher AJ,Gaspar HB (2012) Lentiviral vector mediated gene therapy for X-linked lymphoproliferative disease (XLP1) restores humoral and cellular functions HUMAN GENE THERAPY, 23(5), A8 - A8.
  9. Hassan A,Booth C,Brightwell A,Allwood Z,Veys P,Rao K,Hönig M,Friedrich W,Gennery A,Slatter M,Bredius R,Finocchi A,Cancrini C,Aiuti A,Porta F,Lanfranchi A,Ridella M,Steward C,Filipovich A,Marsh R,Bordon V,Al-Muhsen S,Al-Mousa H,Alsum Z,Al-Dhekri H,Al Ghonaium A,Speckmann C,Fischer A,Mahlaoui N,Nichols KE,Grunebaum E,Al Zahrani D,Roifman CM,Boelens J,Davies EG,Cavazzana-Calvo M,Notarangelo L,Gaspar HB,Inborn Errors Working Party of the European Group for Blood and Marrow Transplantation and European Society for Immunodeficiency (2012) Outcome of hematopoietic stem cell transplantation for adenosine deaminase-deficient severe combined immunodeficiency. Blood, 120(17), 3615 - 3624. 10.1182/blood-2011-12-396879.
  10. Hiwarkar P,Gaspar HB,Gilmour K,Jagani M,Chiesa R,Bennett-Rees N,Breuer J,Rao K,Cale C,Goulden N,Davies G,Amrolia P,Veys P,Qasim W (2012) Impact of viral reactivations in the era of pre-emptive antiviral drug therapy following allogeneic haematopoietic SCT in paediatric recipients. Bone Marrow Transplant. 10.1038/bmt.2012.221.
  11. Engelhardt KR,Gertz EM,Keles S,Schaeffer AA,Ceja R,Sassi A,Massaad MJ,Mellouli F,Benmustapha I,Khemiri M,Kilic SS,Etzioni A,Freeman AF,Thiel J,Schulze I,Al-Herz W,Metin A,Sanal O,Yeganeh M,Niehues T,Siepermann K,Unal E,Patiroglu T,Dasouki M,Yilmaz M,Genel F,Aytekin C,Kutukculer N,Somer A,Kilic M,Reisli I,Camcioglu Y,Gennery AR,Cant AJ,Jones A,Gaspar HB,Arkwright PD,Pietrogrande MC,Baz Z,Al-Tamemi S,Lougaris V,Lefranc G,Megarbane A,Boutros J,Galal N,Bejaoui M,Barbouche R,Geha RS,Chatila TA,Grimbacher B (2012) DOCK8 DEFICIENCY AND A DIAGNOSTIC SCORE TO DIFFERENTIATE IT FROM OTHER HYPER-IGE SYNDROMES JOURNAL OF CLINICAL IMMUNOLOGY, 32, 332 - 333.
  12. Gaspar HB (2012) Gene therapy for ADA-SCID: defining the factors for successful outcome. Blood, 120(18), 3628 - 3629. 10.1182/blood-2012-08-446559.
  13. Booth C,Algar VE,Xu-Bayford J,Fairbanks L,Owens C,Gaspar HB (2012) Non-infectious lung disease in patients with adenosine deaminase deficient severe combined immunodeficiency. J Clin Immunol, 32(3), 449 - 453. 10.1007/s10875-012-9658-3.
  14. Rivat C,Santilli G,Gaspar HB,Thrasher AJ (2012) Gene therapy for primary immunodeficiencies. Hum Gene Ther, 23(7), 668 - 675. 10.1089/hum.2012.116.
  15. Alonso-Ferrero ME,Burke D,Heales S,Thrasher A,Gaspar HB (2011) Gene therapy for lysosomal storage diseases using an enhanced lentiviral vector delivery system HUM GENE THER, 22(10), A44 - A45.
  16. Brown L,Xu-Bayford J,Allwood Z,Slatter M,Cant A,Davies EG,Veys P,Gennery AR,Gaspar HB (2011) Neonatal diagnosis of severe combined immunodeficiency leads to significantly improved survival outcome: the case for newborn screening BLOOD, 117(11), 3243 - 3246. 10.1182/blood-2010-08-300384.
  17. Brown L,Xu-Bayford J,Allwood Z,Slatter M,Cant A,Davies EG,Veys P,Gennery AR,Gaspar HB (2011) Neonatal Diagnosis of Severe Combined Immunodeficiency Leads to Significantly Improved Survival Outcome: The Case for Newborn Screening EDITORIAL COMMENT OBSTET GYNECOL SURV, 66(7), 398 - 399. 10.1097/OGX.0b013e3182338432.
  18. Gholam C,Grigoriadou S,Gilmour KC,Gaspar HB (2011) Familial haemophagocytic lymphohistiocytosis: advances in the genetic basis, diagnosis and management. Clin Exp Immunol, 163(3), 271 - 283. 10.1111/j.1365-2249.2010.04302.x.
  19. Gaspar HB (2011) Induced pluripotent stem cells and primary immunodeficiencies: a new frontier reached, a new world beyond? J Allergy Clin Immunol, 127(6), 1408 - 1409. 10.1016/j.jaci.2011.02.038.
  20. Bacchelli C,Buckland KF,Buckridge S,Salzer U,Schneider P,Thrasher AJ,Gaspar HB (2011) The C76R transmembrane activator and calcium modulator cyclophilin ligand interactor mutation disrupts antibody production and B-cell homeostasis in heterozygous and homozygous mice. J Allergy Clin Immunol, 127(5), 1253 - 9.e13. 10.1016/j.jaci.2011.02.037.
  21. Tolar J,Adair JE,Antoniou M,Bartholomae CC,Becker PS,Blazar BR,Bueren J,Carroll T,Cavazzana-Calvo M,Clapp DW,Dalgleish R,Galy A,Gaspar HB,Hanenberg H,Von Kalle C,Kiem HP,Lindeman D,Naldini L,Navarro S,Renella R,Rio P,Sevilla J,Schmidt M,Verhoeyen E,Wagner JE,Williams DA,Thrasher AJ (2011) Stem Cell Gene Therapy for Fanconi Anemia: Report from the 1st International Fanconi Anemia Gene Therapy Working Group Meeting MOL THER, 19(7), 1193 - 1198. 10.1038/mt.2011.78.
  22. Gaspar HB,Cooray S,Gilmour KC,Parsley KL,Adams S,Howe SJ,Al Ghonaium A,Bayford J,Brown L,Davies EG,Kinnon C,Thrasher AJ (2011) Long-term persistence of a polyclonal T cell repertoire after gene therapy for X-linked severe combined immunodeficiency. Sci Transl Med, 3(97), 97ra79. 10.1126/scitranslmed.3002715.
  23. Gaspar HB,Cooray S,Gilmour KC,Parsley KL,Zhang F,Adams S,Bjorkegren E,Bayford J,Brown L,Davies EG,Veys P,Fairbanks L,Bordon V,Petropoulou T,Kinnon C,Thrasher AJ (2011) Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction. Sci Transl Med, 3(97), 97ra80. 10.1126/scitranslmed.3002716.
  24. Shaw SW,Bollini S,Nader KA,Gastadello A,Mehta V,Filppi E,Cananzi M,Gaspar HB,Qasim W,De Coppi P,David AL (2011) Autologous transplantation of amniotic fluid-derived mesenchymal stem cells into sheep fetuses. Cell Transplant, 20(7), 1015 - 1031. 10.3727/096368910X543402.
  25. Skucek E,Butler S,Gaspar HB,Titman P (2011) Social outcome in children treated by haematopoietic cell transplant for congenital immunodeficiency BONE MARROW TRANSPL, 46(10), 1314 - 1320. 10.1038/bmt.2010.318.
  26. Zhang L,Montiel-Equihua CA,Thrasher AJ,Gaspar HB (2011) Dr Lin Zhang HUMAN GENE THERAPY, 22(10), A53 - A54.
  27. Montiel-Equihua CA,Zhang L,Knight S,Collins M,Saadeh H,Schulz R,Scholz S,Schmidt M,Carmo M,Alonso-Ferrero ME,Blundell MP,Fairbanks LD,Monkeviciute A,Antoniou M,Thrasher AJ,Gaspar HB (2011) Development of a gene expression system for enhanced erythroid expression HUMAN GENE THERAPY, 22(10), A105 - A105.
  28. Cooray S,Gilmour KC,Parsley KL,Zhang F,Bjorkegren E,Bayford J,Brown L,Davies EG,Veys P,Fairbanks L,Bordon V,Petropolou T,Kinnon C,Thrasher AJ,Gaspar HB (2011) Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction HUM GENE THER, 22(10), A40 - A40.
  29. Carmo M,Montiel-Equihua CA,Schambach A,Baum C,Thrasher AJ,Gaspar HB (2011) Development of gene therapy for haemophagocytic lymphohistiocytosis (HLH) due to perforin deficiency HUM GENE THER, 22(10), A46 - A47.
  30. Deichmann A,Brugman MH,Bartholomae CC,Schwarzwaelder K,Verstegen MMA,Howe SJ,Arens A,Ott MG,Hoelzer D,Seger R,Grez M,Hacein-Bey-Abina S,Cavazzana-Calvo M,Fischer A,Paruzynski A,Gabriel R,Glimm H,Abel U,Cattoglio C,Mavilio F,Cassani B,Aiuti A,Dunbar CE,Baum C,Gaspar HB,Thrasher AJ,von Kalle C,Schmidt M,Wagemaker G (2011) Insertion Sites in Engrafted Cells Cluster Within a Limited Repertoire of Genomic Areas After Gammaretroviral Vector Gene Therapy MOL THER, 19(11), 2031 - 2039. 10.1038/mt.2011.178.
  31. Palendira U,Low C,Chan A,Hislop AD,Ho E,Phan TG,Deenick E,Cook MC,Riminton DS,Choo S,Loh R,Alvaro F,Booth C,Gaspar HB,Moretta A,Khanna R,Rickinson AB,Tangye SG (2011) Molecular Pathogenesis of EBV Susceptibility in XLP as Revealed by Analysis of Female Carriers with Heterozygous Expression of SAP PLOS BIOLOGY, 9(11). 10.1371/journal.pbio.1001187.
  32. Booth C,Gaspar HB,Thrasher AJ (2011) Gene therapy for primary immunodeficiency. Curr Opin Pediatr, 23(6), 659 - 666. 10.1097/MOP.0b013e32834cd67a.
  33. Booth C,Gilmour KC,Veys P,Gennery AR,Slatter MA,Chapel H,Heath PT,Steward CG,Smith O,O'Meara A,Kerrigan H,Mahlaoui N,Cavazzana-Calvo M,Fischer A,Moshous D,Blanche S,Pachlopnik Schmid J,Latour S,de Saint-Basile G,Albert M,Notheis G,Rieber N,Strahm B,Ritterbusch H,Lankester A,Hartwig NG,Meyts I,Plebani A,Soresina A,Finocchi A,Pignata C,Cirillo E,Bonanomi S,Peters C,Kalwak K,Pasic S,Sedlacek P,Jazbec J,Kanegane H,Nichols KE,Hanson IC,Kapoor N,Haddad E,Cowan M,Choo S,Smart J,Arkwright PD,Gaspar HB (2011) X-linked lymphoproliferative disease due to SAP/SH2D1A deficiency: a multicenter study on the manifestations, management and outcome of the disease. Blood, 117(1), 53 - 62. 10.1182/blood-2010-06-284935.
  34. Shaw SWS,Bollini S,Nader KA,Gastadello A,Mehta V,Cananzi M,Gaspar HB,Qasim W,De Coppi P,David AL (2010) Wide spread and systemic engraftment after in utero autologous transplantation of amniotic fluid mesenchymal stem cells into sheep fetuses.
  35. Carmo M,Booth C,Montiel-Equihua CA,Schambach A,Baum C,Thrasher AJ,Gaspar HB (2010) Development of gene therapy for haemophagocytic lymphohistiocytosis (HLH) due to perforin deficiency HUMAN GENE THERAPY, 21(10), 1455 - 1455.
  36. Samarasinghe S,Mancao C,Pule M,Nawroly N,Karlsson H,Brewin J,Openshaw P,Gaspar HB,Veys P,Amrolia PJ (2010) Functional characterization of alloreactive T cells identifies CD25 and CD71 as optimal targets for a clinically applicable allodepletion strategy Blood, 115, 396 - 407.
  37. Carmo M,Booth C,Montiel-Equihua CA,Schambach A,Baum C,Thrasher AJ,Gaspar HB (2010) Development of Gene Therapy for HLH Due to Perforin Deficiency HUMAN GENE THERAPY, 21(4), 508 - 509.
  38. Hislop AD,Palendira U,Leese AM,Arkwright PD,Rohrlich PS,Tangye SG,Gaspar HB,Lankester AC,Moretta A,Rickinson AB (2010) Impaired Epstein-Barr virus-specific CD8(+) T-cell function in X-linked lymphoproliferative disease is restricted to SLAM family-positive B-cell targets BLOOD, 116(17), 3249 - 3257. 10.1182/blood-2009-09-238832.
  39. Gaspar HB (2010) Bone Marrow Transplantation and Alternatives for Adenosine Deaminase Deficiency IMMUNOL ALLERGY CLIN, 30(2), 221 - +. 10.1016/j.iac.2010.01.002.
  40. Shaw SWS,Bollini S,Nader KA,Gastadello A,Mehta V,Filppi E,Cananzi M,Gaspar HB,Qasim W,De Coppi P,David AL (2010) Widespread Engraftment after In Utero Autologous Transplantation of Amniotic Fluid Derived Mesenchymal Stem Cells into Sheep Fetuses..
  41. Walshe D,Gaspar HB,Thrasher AJ,Cale CM,Gilmour KC (2010) STAT5 tyrosine phosphorylation for diagnosis and monitoring of patients with severe combined immunodeficiency CLINICAL AND EXPERIMENTAL IMMUNOLOGY, 160, 2 - 2.
  42. Walshe D,Gaspar HB,Thrasher AJ,Cale CM,Gilmour KC (2009) Signal transducer and activator of transcription 5 tyrosine phosphorylation for the diagnosis and monitoring of patients with severe combined immunodeficiency J.Allergy Clin.Immunol., 123(2), 505 - 508.
  43. Antoniou M,Montiel-Equihua C,Monkeviciute A,Thrasher A,Gaspar B (2009) Development of a lentiviral vector for systemic gene therapy of SCID-ADA Human Gene Therapy, 20(11), 1467 - 1468.
  44. Howe SJ,Mansour MR,Schwarzwaelder K,Bartholomae C,Hubank M,Kempski H,Brugman MH,Pike O,K C,S D,Ridder D,Gilmour KC,Adams S,Thornhill SI,Parsley KL,Staal FJ,Gale RE,Linch DC,Bayford J,Brown L,Quaye M,Kinnon C,Ancliff P,Webb DK,Schmidt M,von KC,Gaspar HB,Thrasher AJ (2009) Insertional mutagenesis combined with acquired somatic mutations causes leukaemogenesis following gene therapy of SCID-X1 patients Journal of Clinical Investigation, 118(9), 3143 - 3150.
  45. Gaspar HB,Aiuti A,Porta F,Candotti F,Hershfield MS,Notarangelo LD (2009) How I treat ADA deficiency Blood.
  46. Straathof KC,Rao K,Eyrich M,Hale G,Bird P,Berrie E,Brown L,Adams S,Schlegel PG,Goulden N,Gaspar HB,Gennery AR,Landais P,Davies EG,Brenner MK,Veys PA,Amrolia PJ (2009) Haemopoietic stem-cell transplantation with antibody-based minimal-intensity conditioning: a phase 1/2 study Lancet, 374(9693), 912 - 920.
  47. Qasim W,Gaspar HB,Thrasher AJ (2009) Progress and prospects: gene therapy for inherited immunodeficiencies Gene Ther..
  48. Salzer U,Bacchelli C,Buckridge S,Pan-Hammarstrom Q,Jennings S,Lougaris V,Bergbreiter A,Hagena T,Birmelin J,Plebani A,Webster AD,Peter HH,Suez D,Chapel H,Lean-Tooke A,Spickett GP,nover-Sombke S,Ochs HD,Urschel S,Belohradsky BH,Ugrinovic S,Kumararatne DS,Lawrence TC,Holm AM,Franco JL,Schulze I,Schneider P,Gertz EM,Schaffer AA,Hammarstrom L,Thrasher AJ,Gaspar HB,Grimbacher B (2009) Relevance of biallelic versus monoallelic TNFRSF13B mutations in distinguishing disease-causing from risk-increasing TNFRSF13B variants in antibody deficiency syndromes Blood, 113(9), 1967 - 1976.
  49. Vink CA,Gaspar HB,Gabriel R,Schmidt M,McIvor RS,Thrasher AJ,Qasim W (2009) Sleeping Beauty Transposition From Nonintegrating Lentivirus Molecular Therapy, 17(7), 1197 - 1204.
  50. Straathof KC,Rao K,Eyrich M,Hale G,Bird P,Berrie E,Brown L,Schlegel PG,Goulden N,Gaspar B,Gennery A,Landais P,Davies EG,Brenner MK,Veys P,Amrolia P (2009) A novel antibody-based minimal-intensity conditioning regimen for children with severe organ toxicity or DNA repair disorders Bone Marrow Transplant, 43, S77 - S78.

Qualifications

  • 1989: Bachelor of Medicine/Bachelor of Surgery, King's College London
  • 1986: Bachelor of Science (Honours), King's College London

Keywords

  • ADA SCID
  • Primary immunodeficiency
  • adoptive immunotherapy
  • gene therapy
  • stem cell transplantation