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Items: 1 to 50 of 333

1.

Molecular Evidence of Genome Editing in a Mouse Model of Immunodeficiency.

Abdul-Razak HH, Rocca CJ, Howe SJ, Alonso-Ferrero ME, Wang J, Gabriel R, Bartholomae CC, Gan CHV, Garín MI, Roberts A, Blundell MP, Prakash V, Molina-Estevez FJ, Pantoglou J, Guenechea G, Holmes MC, Gregory PD, Kinnon C, von Kalle C, Schmidt M, Bueren JA, Thrasher AJ, Yáñez-Muñoz RJ.

Sci Rep. 2018 May 29;8(1):8214. doi: 10.1038/s41598-018-26439-9.

2.

WASP-mediated regulation of anti-inflammatory macrophages is IL-10 dependent and is critical for intestinal homeostasis.

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.

Nat Commun. 2018 May 3;9(1):1779. doi: 10.1038/s41467-018-03670-6.

3.

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.

4.

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.

5.

Non-Clinical Efficacy and Safety Studies on G1XCGD, a Lentiviral Vector for Ex Vivo Gene Therapy of X-Linked Chronic Granulomatous Disease.

Brendel C, Rothe M, Santilli G, Charrier S, Stein S, Kunkel H, Abriss D, Müller-Kuller U, Gaspar B, Modlich U, Galy A, Schambach A, Thrasher AJ, Grez M.

Hum Gene Ther Clin Dev. 2018 Jun;29(2):69-79. doi: 10.1089/humc.2017.245. Epub 2018 Apr 17.

PMID:
29664709
6.

Long-Term Hematopoietic Engraftment of Congenic Amniotic Fluid Stem Cells After in Utero Intraperitoneal Transplantation to Immune Competent Mice.

Shangaris P, Loukogeorgakis SP, Blundell MP, Petra E, Shaw SW, Ramachandra DL, Maghsoudlou P, Urbani L, Thrasher AJ, De Coppi P, David AL.

Stem Cells Dev. 2018 Apr 15;27(8):515-523. doi: 10.1089/scd.2017.0116. Epub 2018 Mar 27.

7.

Prevalence and clinical challenges among adults with primary immunodeficiency and recombination-activating gene deficiency.

Lawless D, Geier CB, Farmer JR, Lango Allen H, Thwaites D, Atschekzei F, Brown M, Buchbinder D, Burns SO, Butte MJ, Csomos K, Deevi SVV, Egner W, Ehl S, Eibl MM, Fadugba O, Foldvari Z, Green DM, Henrickson SE, Holland SM, John T, Klemann C, Kuijpers TW, Moreira F, Piller A, Rayner-Matthews P, Romberg ND, Sargur R, Schmidt RE, Schröder C, Schuetz C, Sharapova SO, Smith KGC, Sogkas G, Speckmann C, Stirrups K, Thrasher AJ, Wolf HM, Notarangelo LD, Anwar R, Boyes J, Ujhazi B; NIHR BioResource–Rare Diseases Consortium, Thaventhiran J, Walter JE, Savic S.

J Allergy Clin Immunol. 2018 Jun;141(6):2303-2306. doi: 10.1016/j.jaci.2018.02.007. Epub 2018 Mar 2. No abstract available.

8.

Loss-of-function nuclear factor κB subunit 1 (NFKB1) variants are the most common monogenic cause of common variable immunodeficiency in Europeans.

Tuijnenburg P, Lango Allen H, Burns SO, Greene D, Jansen MH, Staples E, Stephens J, Carss KJ, Biasci D, Baxendale H, Thomas M, Chandra A, Kiani-Alikhan S, Longhurst HJ, Seneviratne SL, Oksenhendler E, Simeoni I, de Bree GJ, Tool ATJ, van Leeuwen EMM, Ebberink EHTM, Meijer AB, Tuna S, Whitehorn D, Brown M, Turro E, Thrasher AJ, Smith KGC, Thaventhiran JE, Kuijpers TW; NIHR BioResource–Rare Diseases Consortium.

J Allergy Clin Immunol. 2018 Mar 2. pii: S0091-6749(18)30286-0. doi: 10.1016/j.jaci.2018.01.039. [Epub ahead of print]

9.

Autonomous role of Wiskott-Aldrich syndrome platelet deficiency in inducing autoimmunity and inflammation.

Sereni L, Castiello MC, Marangoni F, Anselmo A, di Silvestre D, Motta S, Draghici E, Mantero S, Thrasher AJ, Giliani S, Aiuti A, Mauri P, Notarangelo LD, Bosticardo M, Villa A.

J Allergy Clin Immunol. 2018 Feb 6. pii: S0091-6749(18)30201-X. doi: 10.1016/j.jaci.2017.12.1000. [Epub ahead of print]

PMID:
29421274
10.

Leukocyte adhesion deficiency-I: A comprehensive review of all published cases.

Almarza Novoa E, Kasbekar S, Thrasher AJ, Kohn DB, Sevilla J, Nguyen T, Schwartz JD, Bueren JA.

J Allergy Clin Immunol Pract. 2018 Jan 20. pii: S2213-2198(17)31026-7. doi: 10.1016/j.jaip.2017.12.008. [Epub ahead of print] No abstract available.

11.

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 Jan 31. pii: S0091-6749(18)30075-7. doi: 10.1016/j.jaci.2017.11.050. [Epub ahead of print]

12.

Wiskott-Aldrich syndrome protein regulates autophagy and inflammasome activity in innate immune cells.

Lee PP, Lobato-Márquez D, Pramanik N, Sirianni A, Daza-Cajigal V, Rivers E, Cavazza A, Bouma G, Moulding D, Hultenby K, Westerberg LS, Hollinshead M, Lau YL, Burns SO, Mostowy S, Bajaj-Elliott M, Thrasher AJ.

Nat Commun. 2017 Nov 17;8(1):1576. doi: 10.1038/s41467-017-01676-0.

13.

A Personal Reflection from London.

Thrasher AJ.

Hum Gene Ther. 2017 Nov;28(11):959. doi: 10.1089/hum.2017.29052.atr. No abstract available.

PMID:
29035116
14.

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.

15.

Targeted genome editing restores T cell differentiation in a humanized X-SCID pluripotent stem cell disease model.

Alzubi J, Pallant C, Mussolino C, Howe SJ, Thrasher AJ, Cathomen T.

Sci Rep. 2017 Sep 29;7(1):12475. doi: 10.1038/s41598-017-12750-4.

16.

Characterization of a core region in the A2UCOE that confers effective anti-silencing activity.

Zhang F, Santilli G, Thrasher AJ.

Sci Rep. 2017 Aug 31;7(1):10213. doi: 10.1038/s41598-017-10222-3.

17.

Erratum: Lentiviral vectors can be used for full-length dystrophin gene therapy.

Counsell JR, Asgarian Z, Meng J, Ferrer V, Vink CA, Howe SJ, Waddington SN, Thrasher AJ, Muntoni F, Morgan JE, Danos O.

Sci Rep. 2017 Aug 29;7:46880. doi: 10.1038/srep46880.

18.

Wiskott-Aldrich syndrome protein: Emerging mechanisms in immunity.

Rivers E, Thrasher AJ.

Eur J Immunol. 2017 Nov;47(11):1857-1866. doi: 10.1002/eji.201646715. Epub 2017 Sep 12. Review.

PMID:
28805251
19.

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.

PMID:
28716862
20.

Absence of γ-Chain in Keratinocytes Alters Chemokine Secretion, Resulting in Reduced Immune Cell Recruitment.

Nowak K, Linzner D, Thrasher AJ, Lambert PF, Di WL, Burns SO.

J Invest Dermatol. 2017 Oct;137(10):2120-2130. doi: 10.1016/j.jid.2017.05.024. Epub 2017 Jun 17.

PMID:
28634034
21.

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.

22.

Evolving Gene Therapy in Primary Immunodeficiency.

Thrasher AJ, Williams DA.

Mol Ther. 2017 May 3;25(5):1132-1141. doi: 10.1016/j.ymthe.2017.03.018. Epub 2017 Mar 31. Review.

23.

Limiting Thymic Precursor Supply Increases the Risk of Lymphoid Malignancy in Murine X-Linked Severe Combined Immunodeficiency.

Ginn SL, Hallwirth CV, Liao SH, Teber ET, Arthur JW, Wu J, Lee HC, Tay SS, Hu M, Reddel RR, McCormack MP, Thrasher AJ, Cavazzana M, Alexander SI, Alexander IE.

Mol Ther Nucleic Acids. 2017 Mar 17;6:1-14. doi: 10.1016/j.omtn.2016.11.011. Epub 2016 Dec 10.

24.

Lentiviral vectors can be used for full-length dystrophin gene therapy.

Counsell JR, Asgarian Z, Meng J, Ferrer V, Vink CA, Howe SJ, Waddington SN, Thrasher AJ, Muntoni F, Morgan JE, Danos O.

Sci Rep. 2017 Mar 17;7:44775. doi: 10.1038/srep44775. Erratum in: Sci Rep. 2017 Aug 29;7:46880.

25.

Lentiviral vectors can be used for full-length dystrophin gene therapy.

Counsell JR, Asgarian Z, Meng J, Ferrer V, Vink CA, Howe SJ, Waddington SN, Thrasher AJ, Muntoni F, Morgan JE, Danos O.

Sci Rep. 2017 Mar 6;7(1):79. doi: 10.1038/s41598-017-00152-5.

26.

Human Amniocytes Are Receptive to Chemically Induced Reprogramming to Pluripotency.

Hawkins KE, Moschidou D, Faccenda D, Wruck W, Martin-Trujillo A, Hau KL, Ranzoni AM, Sanchez-Freire V, Tommasini F, Eaton S, De Coppi P, Monk D, Campanella M, Thrasher AJ, Adjaye J, Guillot PV.

Mol Ther. 2017 Feb 1;25(2):427-442. doi: 10.1016/j.ymthe.2016.11.014.

27.

Molecular remission of infant B-ALL after infusion of universal TALEN gene-edited CAR T cells.

Qasim W, Zhan H, Samarasinghe S, Adams S, Amrolia P, Stafford S, Butler K, Rivat C, Wright G, Somana K, Ghorashian S, Pinner D, Ahsan G, Gilmour K, Lucchini G, Inglott S, Mifsud W, Chiesa R, Peggs KS, Chan L, Farzeneh F, Thrasher AJ, Vora A, Pule M, Veys P.

Sci Transl Med. 2017 Jan 25;9(374). pii: eaaj2013. doi: 10.1126/scitranslmed.aaj2013. Erratum in: Sci Transl Med. 2017 Feb 15;9(377):null.

PMID:
28123068
28.

A New Chapter on Targeted Gene Insertion for X-CGD: Do Not Skip the Intro(n).

Santilli G, Thrasher AJ.

Mol Ther. 2017 Feb 1;25(2):307-309. doi: 10.1016/j.ymthe.2017.01.002. Epub 2017 Jan 18. No abstract available.

29.

Biallelic JAK1 mutations in immunodeficient patient with mycobacterial infection.

Eletto D, Burns SO, Angulo I, Plagnol V, Gilmour KC, Henriquez F, Curtis J, Gaspar M, Nowak K, Daza-Cajigal V, Kumararatne D, Doffinger R, Thrasher AJ, Nejentsev S.

Nat Commun. 2016 Dec 23;7:13992. doi: 10.1038/ncomms13992.

30.

Autoinflammatory periodic fever, immunodeficiency, and thrombocytopenia (PFIT) caused by mutation in actin-regulatory gene WDR1.

Standing AS, Malinova D, Hong Y, Record J, Moulding D, Blundell MP, Nowak K, Jones H, Omoyinmi E, Gilmour KC, Medlar A, Stanescu H, Kleta R, Anderson G, Nanthapisal S, Gomes SM, Klein N, Eleftheriou D, Thrasher AJ, Brogan PA.

J Exp Med. 2017 Jan;214(1):59-71. doi: 10.1084/jem.20161228. Epub 2016 Dec 19.

31.

Primary immunodeficiencies due to abnormalities of the actin cytoskeleton.

Burns SO, Zarafov A, Thrasher AJ.

Curr Opin Hematol. 2017 Jan;24(1):16-22. Review.

PMID:
27749373
32.

FOXP3+ Tregs require WASP to restrain Th2-mediated food allergy.

Lexmond WS, Goettel JA, Lyons JJ, Jacobse J, Deken MM, Lawrence MG, DiMaggio TH, Kotlarz D, Garabedian E, Sackstein P, Nelson CC, Jones N, Stone KD, Candotti F, Rings EH, Thrasher AJ, Milner JD, Snapper SB, Fiebiger E.

J Clin Invest. 2016 Oct 3;126(10):4030-4044. doi: 10.1172/JCI85129. Epub 2016 Sep 19.

33.

Impact of BREXIT on UK Gene and Cell Therapy: The Need for Continued Pan-European Collaboration.

Baker AH, Ali RR, Thrasher AJ.

Hum Gene Ther. 2016 Sep;27(9):653-5. doi: 10.1089/hum.2016.29033.ahb. No abstract available.

PMID:
27622542
34.

Deletion of Wiskott-Aldrich syndrome protein triggers Rac2 activity and increased cross-presentation by dendritic cells.

Baptista MA, Keszei M, Oliveira M, Sunahara KK, Andersson J, Dahlberg CI, Worth AJ, Liedén A, Kuo IC, Wallin RP, Snapper SB, Eidsmo L, Scheynius A, Karlsson MC, Bouma G, Burns SO, Forsell MN, Thrasher AJ, Nylén S, Westerberg LS.

Nat Commun. 2016 Jul 18;7:12175. doi: 10.1038/ncomms12175.

35.

Automated manufacturing of chimeric antigen receptor T cells for adoptive immunotherapy using CliniMACS prodigy.

Mock U, Nickolay L, Philip B, Cheung GW, Zhan H, Johnston IC, Kaiser AD, Peggs K, Pule M, Thrasher AJ, Qasim W.

Cytotherapy. 2016 Aug;18(8):1002-11. doi: 10.1016/j.jcyt.2016.05.009.

PMID:
27378344
36.

Lentiviral Vector-Mediated Correction of a Mouse Model of Leukocyte Adhesion Deficiency Type I.

Leon-Rico D, Aldea M, Sanchez-Baltasar R, Mesa-Nuñez C, Record J, Burns SO, Santilli G, Thrasher AJ, Bueren JA, Almarza E.

Hum Gene Ther. 2016 Sep;27(9):668-78. doi: 10.1089/hum.2016.016. Epub 2016 May 5.

37.

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
38.

Hyperinflammation in patients with chronic granulomatous disease leads to impairment of hematopoietic stem cell functions.

Weisser M, Demel UM, Stein S, Chen-Wichmann L, Touzot F, Santilli G, Sujer S, Brendel C, Siler U, Cavazzana M, Thrasher AJ, Reichenbach J, Essers MA, Schwäble J, Grez M.

J Allergy Clin Immunol. 2016 Jul;138(1):219-228.e9. doi: 10.1016/j.jaci.2015.11.028. Epub 2016 Feb 4.

PMID:
26853280
39.

Lentiviral Engineered Fibroblasts Expressing Codon-Optimized COL7A1 Restore Anchoring Fibrils in RDEB.

Georgiadis C, Syed F, Petrova A, Abdul-Wahab A, Lwin SM, Farzaneh F, Chan L, Ghani S, Fleck RA, Glover L, McMillan JR, Chen M, Thrasher AJ, McGrath JA, Di WL, Qasim W.

J Invest Dermatol. 2016 Jan;136(1):284-92. doi: 10.1038/JID.2015.364.

40.

Patching up hematopoietic stem cells.

Santilli G, Thrasher AJ.

Nat Biotechnol. 2015 Dec 9;33(12):1236-1238. doi: 10.1038/nbt.3425. No abstract available.

PMID:
26650009
41.

WASp-dependent actin cytoskeleton stability at the dendritic cell immunological synapse is required for extensive, functional T cell contacts.

Malinova D, Fritzsche M, Nowosad CR, Armer H, Munro PM, Blundell MP, Charras G, Tolar P, Bouma G, Thrasher AJ.

J Leukoc Biol. 2016 May;99(5):699-710. doi: 10.1189/jlb.2A0215-050RR. Epub 2015 Nov 20.

42.

Common variable immunodeficiency and natural killer cell lymphopenia caused by Ets-binding site mutation in the IL-2 receptor γ (IL2RG) gene promoter.

Chandra A, Zhang F, Gilmour KC, Webster D, Plagnol V, Kumararatne DS, Burns SO, Nejentsev S, Thrasher AJ.

J Allergy Clin Immunol. 2016 Mar;137(3):940-2.e4. doi: 10.1016/j.jaci.2015.08.049. Epub 2015 Oct 31. No abstract available.

43.

N-WASP is required for B-cell-mediated autoimmunity in Wiskott-Aldrich syndrome.

Volpi S, Santori E, Abernethy K, Mizui M, Dahlberg CI, Recher M, Capuder K, Csizmadia E, Ryan D, Mathew D, Tsokos GC, Snapper S, Westerberg LS, Thrasher AJ, Candotti F, Notarangelo LD.

Blood. 2016 Jan 14;127(2):216-20. doi: 10.1182/blood-2015-05-643817. Epub 2015 Oct 14.

44.

Editorial overview: New technologies.

Baker AH, Thrasher AJ.

Curr Opin Pharmacol. 2015 Oct;24:vii-viii. doi: 10.1016/j.coph.2015.09.005. Epub 2015 Sep 28. No abstract available.

PMID:
26429518
45.

Lentiviral Engineered Fibroblasts Expressing Codon Optimized COL7A1 Restore Anchoring Fibrils in RDEB.

Georgiadis C, Syed F, Petrova A, Abdul-Wahab A, Lwin SM, Farzaneh F, Chan L, Ghani S, Fleck RA, Glover L, McMillan JR, Chen M, Thrasher AJ, McGrath JA, Di WL, Qasim W.

J Invest Dermatol. 2015 Sep 22. doi: 10.1038/jid.2015.364. [Epub ahead of print]

PMID:
26394110
46.

Altered BCR and TLR signals promote enhanced positive selection of autoreactive transitional B cells in Wiskott-Aldrich syndrome.

Kolhatkar NS, Brahmandam A, Thouvenel CD, Becker-Herman S, Jacobs HM, Schwartz MA, Allenspach EJ, Khim S, Panigrahi AK, Luning Prak ET, Thrasher AJ, Notarangelo LD, Candotti F, Torgerson TR, Sanz I, Rawlings DJ.

J Exp Med. 2015 Sep 21;212(10):1663-77. doi: 10.1084/jem.20150585. Epub 2015 Sep 14.

47.

Lentivirus technologies for modulation of the immune system.

Houghton BC, Booth C, Thrasher AJ.

Curr Opin Pharmacol. 2015 Oct;24:119-27. doi: 10.1016/j.coph.2015.08.007. Epub 2015 Sep 10. Review. Erratum in: Curr Opin Pharmacol. 2016 Apr;27:103.

PMID:
26363252
48.

B-cell intrinsic TLR7 signals promote depletion of the marginal zone in a murine model of Wiskott-Aldrich syndrome.

Kolhatkar NS, Scharping NE, Sullivan JM, Jacobs HM, Schwartz MA, Khim S, Notarangelo LD, Thrasher AJ, Rawlings DJ, Jackson SW.

Eur J Immunol. 2015 Oct;45(10):2773-9. doi: 10.1002/eji.201545644. Epub 2015 Aug 31. Erratum in: Eur J Immunol. 2016 Mar;46(3):782.

49.

Immunodeficiency and severe susceptibility to bacterial infection associated with a loss-of-function homozygous mutation of MKL1.

Record J, Malinova D, Zenner HL, Plagnol V, Nowak K, Syed F, Bouma G, Curtis J, Gilmour K, Cale C, Hackett S, Charras G, Moulding D, Nejentsev S, Thrasher AJ, Burns SO.

Blood. 2015 Sep 24;126(13):1527-35. doi: 10.1182/blood-2014-12-611012. Epub 2015 Jul 29.

50.

Current and emerging treatment options for Wiskott-Aldrich syndrome.

Worth AJ, Thrasher AJ.

Expert Rev Clin Immunol. 2015;11(9):1015-32. doi: 10.1586/1744666X.2015.1062366. Epub 2015 Jul 9. Review.

PMID:
26159751

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