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Items: 1 to 50 of 167

1.

Universal Newborn Screening for Severe Combined Immunodeficiency (SCID).

van der Burg M, Mahlaoui N, Gaspar HB, Pai SY.

Front Pediatr. 2019 Sep 18;7:373. doi: 10.3389/fped.2019.00373. eCollection 2019. Review.

2.

Pre-clinical Safety and Efficacy of Lentiviral Vector-Mediated Ex Vivo Stem Cell Gene Therapy for the Treatment of Mucopolysaccharidosis IIIA.

Ellison SM, Liao A, Wood S, Taylor J, Youshani AS, Rowlston S, Parker H, Armant M, Biffi A, Chan L, Farzaneh F, Wynn R, Jones SA, Heal P, Gaspar HB, Bigger BW.

Mol Ther Methods Clin Dev. 2019 Apr 6;13:399-413. doi: 10.1016/j.omtm.2019.04.001. eCollection 2019 Jun 14.

3.

Clinical and immunological features in a cohort of patients with partial DiGeorge syndrome followed at a single center.

Giardino G, Radwan N, Koletsi P, Morrogh DM, Adams S, Ip W, Worth A, Jones A, Meyer-Parsonson I, Gaspar HB, Gilmour K, Davies EG, Ladomenou F.

Blood. 2019 Jun 13;133(24):2586-2596. doi: 10.1182/blood.2018885244. Epub 2019 Apr 23.

PMID:
31015189
4.

Hematopoietic Stem Cell Transplantation as Treatment for Patients with DOCK8 Deficiency.

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, Doğu 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; Inborn Errors Working Party of the European Group for Blood and Marrow Transplantation and the European Society for Primary Immunodeficiencies.

J Allergy Clin Immunol Pract. 2019 Mar;7(3):848-855. doi: 10.1016/j.jaip.2018.10.035. Epub 2018 Nov 2.

PMID:
30391550
5.

Consensus approach for the management of severe combined immune deficiency caused by adenosine deaminase deficiency.

Kohn DB, Hershfield MS, Puck JM, Aiuti A, Blincoe A, Gaspar HB, Notarangelo LD, Grunebaum E.

J Allergy Clin Immunol. 2019 Mar;143(3):852-863. doi: 10.1016/j.jaci.2018.08.024. Epub 2018 Sep 5.

PMID:
30194989
6.

Preclinical Development of a Lentiviral Vector for Gene Therapy of X-Linked Severe Combined Immunodeficiency.

Poletti V, Charrier S, Corre G, Gjata B, Vignaud A, Zhang F, Rothe M, Schambach A, Gaspar HB, Thrasher AJ, Mavilio F.

Mol Ther Methods Clin Dev. 2018 Mar 10;9:257-269. doi: 10.1016/j.omtm.2018.03.002. eCollection 2018 Jun 15.

7.

Transfer of gene-corrected T cells corrects humoral and cytotoxic defects in patients with X-linked lymphoproliferative disease.

Panchal N, Houghton B, Diez B, Ghosh S, Ricciardelli I, Thrasher AJ, Gaspar HB, Booth C.

J Allergy Clin Immunol. 2018 Jul;142(1):235-245.e6. doi: 10.1016/j.jaci.2018.02.053. Epub 2018 Apr 27.

8.

Non-posttransplant lymphoproliferative disorder malignancy after hematopoietic stem cell transplantation in patients with primary immunodeficiency: UK experience.

Unni MNM, Elfeky R, Rao K, Nademi Z, Chiesa R, Amrolia P, Skinner R, Slater O, Worth A, Flood T, Abinun M, Hambleton S, Qasim W, Gaspar HB, Cant AJ, Gennery AR, Veys P, Slatter MA.

J Allergy Clin Immunol. 2018 Jun;141(6):2319-2321.e1. doi: 10.1016/j.jaci.2018.02.038. Epub 2018 Mar 7. No abstract available.

PMID:
29524534
9.

T-cell gene therapy for perforin deficiency corrects cytotoxicity defects and prevents hemophagocytic lymphohistiocytosis manifestations.

Ghosh S, Carmo M, Calero-Garcia M, Ricciardelli I, Bustamante Ogando JC, Blundell MP, Schambach A, Ashton-Rickardt PG, Booth C, Ehl S, Lehmberg K, Thrasher AJ, Gaspar HB.

J Allergy Clin Immunol. 2018 Sep;142(3):904-913.e3. doi: 10.1016/j.jaci.2017.11.050. Epub 2018 Jan 31.

10.

Recent advances in understanding the pathogenesis and management of reticular dysgenesis.

Hoenig M, Pannicke U, Gaspar HB, Schwarz K.

Br J Haematol. 2018 Mar;180(5):644-653. doi: 10.1111/bjh.15045. Epub 2017 Dec 21. Review.

PMID:
29270983
11.

Treosulfan and Fludarabine Conditioning for Hematopoietic Stem Cell Transplantation in Children with Primary Immunodeficiency: UK Experience.

Slatter MA, Rao K, Abd Hamid IJ, Nademi Z, Chiesa R, Elfeky R, Pearce MS, Amrolia P, Worth A, Flood T, Abinun M, Hambleton S, Qasim W, Gaspar HB, Cant AJ, Gennery AR, Veys P.

Biol Blood Marrow Transplant. 2018 Mar;24(3):529-536. doi: 10.1016/j.bbmt.2017.11.009. Epub 2017 Nov 16.

12.

Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy.

Eichler F, Duncan C, Musolino PL, Orchard PJ, De Oliveira S, Thrasher AJ, Armant M, Dansereau C, Lund TC, Miller WP, Raymond GV, Sankar R, Shah AJ, Sevin C, Gaspar HB, Gissen P, Amartino H, Bratkovic D, Smith NJC, Paker AM, Shamir E, O'Meara T, Davidson D, Aubourg P, Williams DA.

N Engl J Med. 2017 Oct 26;377(17):1630-1638. doi: 10.1056/NEJMoa1700554. Epub 2017 Oct 4.

13.

Gene Therapy Approaches to Immunodeficiency.

Ghosh S, Gaspar HB.

Hematol Oncol Clin North Am. 2017 Oct;31(5):823-834. doi: 10.1016/j.hoc.2017.05.003. Epub 2017 Jun 29. Review.

PMID:
28895850
14.

Adenosine Deaminase (ADA)-Deficient Severe Combined Immune Deficiency (SCID): Molecular Pathogenesis and Clinical Manifestations.

Bradford KL, Moretti FA, Carbonaro-Sarracino DA, Gaspar HB, Kohn DB.

J Clin Immunol. 2017 Oct;37(7):626-637. doi: 10.1007/s10875-017-0433-3. Epub 2017 Aug 25. Review.

PMID:
28842866
15.

Gene therapy for Wiskott-Aldrich syndrome in a severely affected adult.

Morris EC, Fox T, Chakraverty R, Tendeiro R, Snell K, Rivat C, Grace S, Gilmour K, Workman S, Buckland K, Butler K, Chee R, Salama AD, Ibrahim H, Hara H, Duret C, Mavilio F, Male F, Bushman FD, Galy A, Burns SO, Gaspar HB, Thrasher AJ.

Blood. 2017 Sep 14;130(11):1327-1335. doi: 10.1182/blood-2017-04-777136. Epub 2017 Jul 17.

16.

Lentiviral Vectors with Cellular Promoters Correct Anemia and Lethal Bone Marrow Failure in a Mouse Model for Diamond-Blackfan Anemia.

Debnath S, Jaako P, Siva K, Rothe M, Chen J, Dahl M, Gaspar HB, Flygare J, Schambach A, Karlsson S.

Mol Ther. 2017 Aug 2;25(8):1805-1814. doi: 10.1016/j.ymthe.2017.04.002. Epub 2017 Apr 20.

17.

Thymus transplantation for complete DiGeorge syndrome: European experience.

Davies EG, Cheung M, Gilmour K, Maimaris J, Curry J, Furmanski A, Sebire N, Halliday N, Mengrelis K, Adams S, Bernatoniene J, Bremner R, Browning M, Devlin B, Erichsen HC, Gaspar HB, Hutchison L, Ip W, Ifversen M, Leahy TR, McCarthy E, Moshous D, Neuling K, Pac M, Papadopol A, Parsley KL, Poliani L, Ricciardelli I, Sansom DM, Voor T, Worth A, Crompton T, Markert ML, Thrasher AJ.

J Allergy Clin Immunol. 2017 Dec;140(6):1660-1670.e16. doi: 10.1016/j.jaci.2017.03.020. Epub 2017 Apr 8.

18.

Outcome of hematopoietic cell transplantation for DNA double-strand break repair disorders.

Slack J, Albert MH, Balashov D, Belohradsky BH, Bertaina A, Bleesing J, Booth C, Buechner J, Buckley RH, Ouachée-Chardin M, Deripapa E, Drabko K, Eapen M, Feuchtinger T, Finocchi A, Gaspar HB, Ghosh S, Gillio A, Gonzalez-Granado LI, Grunebaum E, Güngör T, Heilmann C, Helminen M, Higuchi K, Imai K, Kalwak K, Kanazawa N, Karasu G, Kucuk ZY, Laberko A, Lange A, Mahlaoui N, Meisel R, Moshous D, Muramatsu H, Parikh S, Pasic S, Schmid I, Schuetz C, Schulz A, Schultz KR, Shaw PJ, Slatter MA, Sykora KW, Tamura S, Taskinen M, Wawer A, Wolska-Kuśnierz B, Cowan MJ, Fischer A, Gennery AR; Inborn Errors Working Party of the European Society for Blood and Marrow Transplantation and the European Society for Immunodeficiencies; Stem Cell Transplant for Immunodeficiencies in Europe (SCETIDE); Center for International Blood and Marrow Transplant Research; Primary Immunodeficiency Treatment Consortium.

J Allergy Clin Immunol. 2018 Jan;141(1):322-328.e10. doi: 10.1016/j.jaci.2017.02.036. Epub 2017 Apr 7.

19.

Reticular dysgenesis: international survey on clinical presentation, transplantation, and outcome.

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; European Society for Blood and Marrow Transplantation (EBMT) Inborn Errors Working Party.

Blood. 2017 May 25;129(21):2928-2938. doi: 10.1182/blood-2016-11-745638. Epub 2017 Mar 22.

20.

How We Manage Adenosine Deaminase-Deficient Severe Combined Immune Deficiency (ADA SCID).

Kohn DB, Gaspar HB.

J Clin Immunol. 2017 May;37(4):351-356. doi: 10.1007/s10875-017-0373-y. Epub 2017 Feb 14. Review.

PMID:
28194615
21.

A prospective study on the natural history of patients with profound combined immunodeficiency: An interim analysis.

Speckmann C, Doerken S, Aiuti A, Albert MH, Al-Herz W, Allende LM, Scarselli A, Avcin T, Perez-Becker R, Cancrini C, Cant A, Di Cesare S, Finocchi A, Fischer A, Gaspar HB, Ghosh S, Gennery A, Gilmour K, González-Granado LI, Martinez-Gallo M, Hambleton S, Hauck F, Hoenig M, Moshous D, Neven B, Niehues T, Notarangelo L, Picard C, Rieber N, Schulz A, Schwarz K, Seidel MG, Soler-Palacin P, Stepensky P, Strahm B, Vraetz T, Warnatz K, Winterhalter C, Worth A, Fuchs S, Uhlmann A, Ehl S; P-CID study of the Inborn Errors Working Party of the EBMT.

J Allergy Clin Immunol. 2017 Apr;139(4):1302-1310.e4. doi: 10.1016/j.jaci.2016.07.040. Epub 2016 Sep 19.

22.

Adenosine Deaminase Deficiency - More Than Just an Immunodeficiency.

Whitmore KV, Gaspar HB.

Front Immunol. 2016 Aug 16;7:314. doi: 10.3389/fimmu.2016.00314. eCollection 2016. Review.

23.

Mutations in linker for activation of T cells (LAT) lead to a novel form of severe combined immunodeficiency.

Bacchelli C, Moretti FA, Carmo M, Adams S, Stanescu HC, Pearce K, Madkaikar M, Gilmour KC, Nicholas AK, Woods CG, Kleta R, Beales PL, Qasim W, Gaspar HB.

J Allergy Clin Immunol. 2017 Feb;139(2):634-642.e5. doi: 10.1016/j.jaci.2016.05.036. Epub 2016 Jul 15.

PMID:
27522155
24.

Hematopoietic stem cell transplantation outcomes for 11 patients with dedicator of cytokinesis 8 deficiency.

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.

J Allergy Clin Immunol. 2016 Sep;138(3):852-859.e3. doi: 10.1016/j.jaci.2016.02.022. Epub 2016 Apr 6.

25.

A novel Rab27a mutation binds melanophilin, but not Munc13-4, causing immunodeficiency without albinism.

Netter P, Chan SK, Banerjee PP, Monaco-Shawver L, Noroski LM, Hanson IC, Forbes LR, Mace EM, Chinen J, Gaspar HB, Sleiman P, Hakonarson H, Klein C, Ehlayel MS, Orange JS.

J Allergy Clin Immunol. 2016 Aug;138(2):599-601.e3. doi: 10.1016/j.jaci.2015.12.1337. Epub 2016 Mar 23. No abstract available.

26.

Treating Immunodeficiency through HSC Gene Therapy.

Booth C, Gaspar HB, Thrasher AJ.

Trends Mol Med. 2016 Apr;22(4):317-327. doi: 10.1016/j.molmed.2016.02.002. Epub 2016 Mar 15. Review.

PMID:
26993219
27.

Erratum to: Adenosine Deaminase Deficient Severe Combined Immunodeficiency Presenting as Atypical Haemolytic Uraemic Syndrome.

Nikolajeva O, Worth A, Hague R, Martinez-Alier N, Smart J, Adams S, Davies EG, Gaspar HB.

J Clin Immunol. 2016 May;36(4):413. No abstract available.

PMID:
26951489
28.

Autologous Transplantation of Amniotic Fluid-Derived Mesenchymal Stem Cells into Sheep Fetuses.

Shaw SWS, Bollini S, Nader KA, Gastaldello A, Mehta V, Filppi E, Cananzi M, Gaspar HB, Qasim W, De Coppi P, David AL.

Cell Transplant. 2016 Mar;25(3):615. doi: 10.3727/096368916X691006.

PMID:
28836829
29.

Combined immunodeficiency due to JAK3 mutation in a child presenting with skin granuloma.

Scarselli A, Di Cesare S, Di Matteo G, De Matteis A, Ariganello P, Romiti ML, Cascioli S, De Vito R, Bertaina A, Locatelli F, Gaspar HB, Aiuti A, Rossi P, Gilmour K, Cancrini C.

J Allergy Clin Immunol. 2016 Mar;137(3):948-51.e5. doi: 10.1016/j.jaci.2015.09.017. Epub 2015 Nov 3. No abstract available.

PMID:
26545580
30.

Primary Immunodeficiency Diseases: an Update on the Classification from the International Union of Immunological Societies Expert Committee for Primary Immunodeficiency 2015.

Picard C, Al-Herz W, Bousfiha A, Casanova JL, Chatila T, Conley ME, Cunningham-Rundles C, Etzioni A, Holland SM, Klein C, Nonoyama S, Ochs HD, Oksenhendler E, Puck JM, Sullivan KE, Tang ML, Franco JL, Gaspar HB.

J Clin Immunol. 2015 Nov;35(8):696-726. doi: 10.1007/s10875-015-0201-1. Epub 2015 Oct 19. Review.

31.

The 2015 IUIS Phenotypic Classification for Primary Immunodeficiencies.

Bousfiha A, Jeddane L, Al-Herz W, Ailal F, Casanova JL, Chatila T, Conley ME, Cunningham-Rundles C, Etzioni A, Franco JL, Gaspar HB, Holland SM, Klein C, Nonoyama S, Ochs HD, Oksenhendler E, Picard C, Puck JM, Sullivan KE, Tang ML.

J Clin Immunol. 2015 Nov;35(8):727-38. doi: 10.1007/s10875-015-0198-5. Epub 2015 Oct 7. Review.

32.

Chronic Infection with Rotavirus Vaccine Strains in UK Children with Severe Combined Immunodeficiency.

Morillo-Gutierrez B, Worth A, Valappil M, Gaspar HB, Gennery AR.

Pediatr Infect Dis J. 2015 Sep;34(9):1040-1. doi: 10.1097/INF.0000000000000788. No abstract available.

PMID:
26376312
33.

Gene therapy for monogenic disorders of the bone marrow.

Ghosh S, Thrasher AJ, Gaspar HB.

Br J Haematol. 2015 Oct;171(2):155-170. doi: 10.1111/bjh.13520. Epub 2015 Jun 5. Review.

PMID:
26044877
34.

Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome.

Hacein-Bey Abina S, Gaspar HB, Blondeau J, Caccavelli L, Charrier S, Buckland K, Picard C, Six E, Himoudi N, Gilmour K, McNicol AM, Hara H, Xu-Bayford J, Rivat C, Touzot F, Mavilio F, Lim A, Treluyer JM, Héritier S, Lefrère F, Magalon J, Pengue-Koyi I, Honnet G, Blanche S, Sherman EA, Male F, Berry C, Malani N, Bushman FD, Fischer A, Thrasher AJ, Galy A, Cavazzana M.

JAMA. 2015 Apr 21;313(15):1550-63. doi: 10.1001/jama.2015.3253.

35.

Adenosine deaminase deficient severe combined immunodeficiency presenting as atypical haemolytic uraemic syndrome.

Nikolajeva O, Worth A, Hague R, Martinez-Alier N, Smart J, Adams S, Davies EG, Gaspar HB.

J Clin Immunol. 2015 May;35(4):366-72. doi: 10.1007/s10875-015-0158-0. Epub 2015 Apr 15. Erratum in: J Clin Immunol. 2016 May;36(4):413.

PMID:
25875700
36.

Variable phenotype of severe immunodeficiencies associated with RMRP gene mutations.

Ip W, Gaspar HB, Kleta R, Chanudet E, Bacchelli C, Pitts A, Nademi Z, Davies EG, Slatter MA, Amrolia P, Rao K, Veys P, Gennery AR, Qasim W.

J Clin Immunol. 2015 Feb;35(2):147-57. doi: 10.1007/s10875-015-0135-7. Epub 2015 Feb 8.

PMID:
25663137
37.

DOCK8 deficiency: clinical and immunological phenotype and treatment options - a review of 136 patients.

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; inborn errors working party of EBMT.

J Clin Immunol. 2015 Feb;35(2):189-98. doi: 10.1007/s10875-014-0126-0. Epub 2015 Jan 28.

PMID:
25627830
38.

Site- and allele-specific polycomb dysregulation in T-cell leukaemia.

Navarro JM, Touzart A, Pradel LC, Loosveld M, Koubi M, Fenouil R, Le Noir S, Maqbool MA, Morgado E, Gregoire C, Jaeger S, Mamessier E, Pignon C, Hacein-Bey-Abina S, Malissen B, Gut M, Gut IG, Dombret H, Macintyre EA, Howe SJ, Gaspar HB, Thrasher AJ, Ifrah N, Payet-Bornet D, Duprez E, Andrau JC, Asnafi V, Nadel B.

Nat Commun. 2015 Jan 23;6:6094. doi: 10.1038/ncomms7094.

39.

Multicenter experience in hematopoietic stem cell transplantation for serious complications of common variable immunodeficiency.

Wehr C, Gennery AR, Lindemans C, Schulz A, Hoenig M, Marks R, Recher M, Gruhn B, Holbro A, Heijnen I, Meyer D, Grigoleit G, Einsele H, Baumann U, Witte T, Sykora KW, Goldacker S, Regairaz L, Aksoylar S, Ardeniz Ö, Zecca M, Zdziarski P, Meyts I, Matthes-Martin S, Imai K, Kamae C, Fielding A, Seneviratne S, Mahlaoui N, Slatter MA, Güngör T, Arkwright PD, van Montfrans J, Sullivan KE, Grimbacher B, Cant A, Peter HH, Finke J, Gaspar HB, Warnatz K, Rizzi M; Inborn Errors Working Party of the European Society for Blood and Marrow Transplantation and the European Society for Immunodeficiency.

J Allergy Clin Immunol. 2015 Apr;135(4):988-97.e6. doi: 10.1016/j.jaci.2014.11.029. Epub 2015 Jan 14.

PMID:
25595268
40.

Primary immune deficiencies - principles of care.

Chapel H, Prevot J, Gaspar HB, Español T, Bonilla FA, Solis L, Drabwell J; Editorial Board for Working Party on Principles of Care at IPOPI.

Front Immunol. 2014 Dec 15;5:627. doi: 10.3389/fimmu.2014.00627. eCollection 2014. Review.

41.

Severe combined immunodeficiency: recent developments and guidance on clinical management.

Rivers L, Gaspar HB.

Arch Dis Child. 2015 Jul;100(7):667-72. doi: 10.1136/archdischild-2014-306425. Epub 2015 Jan 6. Review.

PMID:
25564533
42.

Perforin gene transfer into hematopoietic stem cells improves immune dysregulation in murine models of perforin deficiency.

Carmo M, Risma KA, Arumugam P, Tiwari S, Hontz AE, Montiel-Equihua CA, Alonso-Ferrero ME, Blundell MP, Schambach A, Baum C, Malik P, Thrasher AJ, Jordan MB, Gaspar HB.

Mol Ther. 2015 Apr;23(4):737-45. doi: 10.1038/mt.2014.242. Epub 2014 Dec 19.

43.

Gene-ectomy: gene ablation with CRISPR/Cas9 in human hematopoietic cells.

Calero-Garcia M, Gaspar HB.

Cell Stem Cell. 2014 Nov 6;15(5):529-30. doi: 10.1016/j.stem.2014.10.014. Epub 2014 Nov 6.

44.

A modified γ-retrovirus vector for X-linked severe combined immunodeficiency.

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.

N Engl J Med. 2014 Oct 9;371(15):1407-17. doi: 10.1056/NEJMoa1404588.

45.

Gene therapy for haemophagocytic lymphohistiocytosis.

Booth C, Carmo M, Gaspar HB.

Curr Gene Ther. 2014;14(6):437-46. Review.

PMID:
25245087
46.

"Darwinian" tumor-suppression model unsupported in clinical experience.

Qasim W, Gaspar HB, Thrasher AJ.

Mol Ther. 2014 Sep;22(9):1562-3. doi: 10.1038/mt.2014.147. No abstract available.

47.

Successful RAG1-SCID gene therapy depends on the level of RAG1 expression.

Pike-Overzet K, Baum C, Bredius RG, Cavazzana M, Driessen GJ, Fibbe WE, Gaspar HB, Hoeben RC, Lagresle-Peyrou C, Lankester A, Meij P, Schambach A, Thrasher A, Van Dongen JJ, Zwaginga JJ, Staal FJ.

J Allergy Clin Immunol. 2014 Jul;134(1):242-3. doi: 10.1016/j.jaci.2014.04.033. Epub 2014 Jun 26. No abstract available.

PMID:
25117803
48.

Primary immunodeficiency diseases: an update on the classification from the international union of immunological societies expert committee for primary immunodeficiency.

Al-Herz W, Bousfiha A, Casanova JL, Chatila T, Conley ME, Cunningham-Rundles C, Etzioni A, Franco JL, Gaspar HB, Holland SM, Klein C, Nonoyama S, Ochs HD, Oksenhendler E, Picard C, Puck JM, Sullivan K, Tang ML.

Front Immunol. 2014 Apr 22;5:162. doi: 10.3389/fimmu.2014.00162. eCollection 2014. Erratum in: Front Immunol. 2014;5:460.

49.

The case for mandatory newborn screening for severe combined immunodeficiency (SCID).

Gaspar HB, Hammarström L, Mahlaoui N, Borte M, Borte S.

J Clin Immunol. 2014 May;34(4):393-7. doi: 10.1007/s10875-014-0029-0. Epub 2014 Apr 2. Review.

PMID:
24691999
50.

Immunodeficiency and disseminated mycobacterial infection associated with homozygous nonsense mutation of IKKβ.

Burns SO, Plagnol V, Gutierrez BM, Al Zahrani D, Curtis J, Gaspar M, Hassan A, Jones AM, Malone M, Rampling D, McLatchie A, Doffinger R, Gilmour KC, Henriquez F, Thrasher AJ, Gaspar HB, Nejentsev S.

J Allergy Clin Immunol. 2014 Jul;134(1):215-8. doi: 10.1016/j.jaci.2013.12.1093. Epub 2014 Mar 27. No abstract available.

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