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Items: 1 to 50 of 357

1.

Clonal tracking in gene therapy patients reveals a diversity of human hematopoietic differentiation programs.

Six E, Guilloux A, Denis A, Lecoules A, Magnani A, Vilette R, Male F, Cagnard N, Delville M, Magrin E, Caccavelli L, Roudaut C, Plantier C, Sobrino S, Gregg J, Nobles CL, Everett JK, Hacein-Bey-Abina S, Galy A, Fischer A, Thrasher AJ, André I, Cavazzana M, Bushman F.

Blood. 2020 Feb 7. pii: blood.2019002350. doi: 10.1182/blood.2019002350. [Epub ahead of print]

PMID:
32040546
2.

High throughput <i>in vitro</i>, <i>ex vivo</i> and i<i>n vivo</i> screen of AAV vectors based on physical and functional transduction.

Westhaus A, Cabanes Creus M, Rybicki A, Baltazar G, Navarro RG, Zhu E, Drouyer M, Knight M, Albu RF, Ng BH, Kalajdzic P, Kwiatek M, Hsu K, Santilli G, Gold W, Kramer B, Gonzalez-Cordero A, Thrasher AJ, Alexander IE, Lisowski L.

Hum Gene Ther. 2020 Jan 30. doi: 10.1089/hum.2019.264. [Epub ahead of print]

PMID:
32000541
3.

Lentiviral gene therapy for X-linked chronic granulomatous disease.

Kohn DB, Booth C, Kang EM, Pai SY, Shaw KL, Santilli G, Armant M, Buckland KF, Choi U, De Ravin SS, Dorsey MJ, Kuo CY, Leon-Rico D, Rivat C, Izotova N, Gilmour K, Snell K, Dip JX, Darwish J, Morris EC, Terrazas D, Wang LD, Bauser CA, Paprotka T, Kuhns DB, Gregg J, Raymond HE, Everett JK, Honnet G, Biasco L, Newburger PE, Bushman FD, Grez M, Gaspar HB, Williams DA, Malech HL, Galy A, Thrasher AJ; Net4CGD consortium.

Nat Med. 2020 Feb;26(2):200-206. doi: 10.1038/s41591-019-0735-5. Epub 2020 Jan 27.

PMID:
31988463
4.

Lentiviral Vector Production Titer Is Not Limited in HEK293T by Induced Intracellular Innate Immunity.

Ferreira CB, Sumner RP, Rodriguez-Plata MT, Rasaiyaah J, Milne RS, Thrasher AJ, Qasim W, Towers GJ.

Mol Ther Methods Clin Dev. 2019 Dec 24;17:209-219. doi: 10.1016/j.omtm.2019.11.021. eCollection 2020 Jun 12.

5.

Publisher Correction: In Utero Gene Therapy (IUGT) Using GLOBE Lentiviral Vector Phenotypically Corrects the Heterozygous Humanised Mouse Model and Its Progress Can Be Monitored Using MRI Techniques.

Shangaris P, Loukogeorgakis SP, Subramaniam S, Flouri C, Jackson LH, Wang W, Blundell MP, Liu S, Eaton S, Bakhamis N, Ramachandra DL, Maghsoudlou P, Urbani L, Waddington SN, Eddaoudi A, Archer J, Antoniou MN, Stuckey DJ, Schmidt M, Thrasher AJ, Ryan TM, De Coppi P, David AL.

Sci Rep. 2019 Dec 24;9(1):20214. doi: 10.1038/s41598-019-55754-y.

6.

Differential Transgene Silencing of Myeloid-Specific Promoters in the AAVS1 Safe Harbor Locus of Induced Pluripotent Stem Cell-Derived Myeloid Cells.

Klatt D, Cheng E, Hoffmann D, Santilli G, Thrasher AJ, Brendel C, Schambach A.

Hum Gene Ther. 2020 Jan 23. doi: 10.1089/hum.2019.194. [Epub ahead of print]

PMID:
31773990
7.

Correction of both immunodeficiency and hypoparathyroidism by thymus transplantation in complete DiGeorge syndrome.

Kreins AY, Junghanns F, Mifsud W, Somana K, Sebire N, Rampling D, Worth A, Sirin M, Schuetz C, Schulz A, Hoenig M, Thrasher AJ, Davies EG.

Am J Transplant. 2019 Oct 30. doi: 10.1111/ajt.15668. [Epub ahead of print]

PMID:
31663273
8.

An intronic deletion in megakaryoblastic leukemia 1 is associated with hyperproliferation of B cells in triplets with Hodgkin lymphoma.

Record J, Sendel A, Kritikou JS, Kuznetsov NV, Brauner H, He M, Nagy N, Oliveira MMS, Griseti E, Haase CB, Dahlström J, Boddul S, Wermeling F, Thrasher AJ, Liu C, Andersson J, Claesson HE, Winqvist O, Burns SO, Björkholm M, Westerberg LS.

Haematologica. 2019 Oct 3. pii: haematol.2019.216317. doi: 10.3324/haematol.2019.216317. [Epub ahead of print]

9.

Loss of Janus Associated Kinase 1 Alters Urothelial Cell Function and Facilitates the Development of Bladder Cancer.

Daza-Cajigal V, Albuquerque AS, Pearson J, Hinley J, Mason AS, Stahlschmidt J, Thrasher AJ, Mishra V, Southgate J, Burns SO.

Front Immunol. 2019 Sep 10;10:2065. doi: 10.3389/fimmu.2019.02065. eCollection 2019.

10.

Targeted Repair of p47-CGD in iPSCs by CRISPR/Cas9: Functional Correction without Cleavage in the Highly Homologous Pseudogenes.

Klatt D, Cheng E, Philipp F, Selich A, Dahlke J, Schmidt RE, Schott JW, Büning H, Hoffmann D, Thrasher AJ, Schambach A.

Stem Cell Reports. 2019 Oct 8;13(4):590-598. doi: 10.1016/j.stemcr.2019.08.008. Epub 2019 Sep 19.

11.

In Utero Gene Therapy (IUGT) Using GLOBE Lentiviral Vector Phenotypically Corrects the Heterozygous Humanised Mouse Model and Its Progress Can Be Monitored Using MRI Techniques.

Shangaris P, Loukogeorgakis SP, Subramaniam S, Flouri C, Jackson LH, Wang W, Blundell MP, Liu S, Eaton S, Bakhamis N, Ramachandra DL, Maghsoudlou P, Urbani L, Waddington SN, Eddaoudi A, Archer J, Antoniou MN, Stuckey DJ, Schmidt M, Thrasher AJ, Ryan TM, De Coppi P, David AL.

Sci Rep. 2019 Aug 12;9(1):11592. doi: 10.1038/s41598-019-48078-4. Erratum in: Sci Rep. 2019 Dec 24;9(1):20214.

12.

Enhancing Lentiviral and Alpharetroviral Transduction of Human Hematopoietic Stem Cells for Clinical Application.

Schott JW, León-Rico D, Ferreira CB, Buckland KF, Santilli G, Armant MA, Schambach A, Cavazza A, Thrasher AJ.

Mol Ther Methods Clin Dev. 2019 Jun 7;14:134-147. doi: 10.1016/j.omtm.2019.05.015. eCollection 2019 Sep 13.

13.

Gene therapy for primary immunodeficiency.

Booth C, Romano R, Roncarolo MG, Thrasher AJ.

Hum Mol Genet. 2019 Oct 1;28(R1):R15-R23. doi: 10.1093/hmg/ddz170.

PMID:
31297531
14.

Generation and Clinical Application of Gene-Modified Autologous Epidermal Sheets in Netherton Syndrome: Lessons Learned from a Phase 1 Trial.

Di WL, Lwin SM, Petrova A, Bernadis C, Syed F, Farzaneh F, Moulding D, Martinez AE, Sebire NJ, Rampling D, Virasami A, Zamiri M, Wang W, Hara H, Kadiyirire T, Abdul-Wahab A, Martinez-Queipo M, Harper JI, McGrath JA, Thrasher AJ, Mellerio JE, Qasim W.

Hum Gene Ther. 2019 Sep;30(9):1067-1078. doi: 10.1089/hum.2019.049. Epub 2019 Aug 5.

PMID:
31288584
15.

Loss of the interleukin-6 receptor causes immunodeficiency, atopy, and abnormal inflammatory responses.

Spencer S, Köstel Bal S, Egner W, Lango Allen H, Raza SI, Ma CA, Gürel M, Zhang Y, Sun G, Sabroe RA, Greene D, Rae W, Shahin T, Kania K, Ardy RC, Thian M, Staples E, Pecchia-Bekkum A, Worrall WPM, Stephens J, Brown M, Tuna S, York M, Shackley F, Kerrin D, Sargur R, Condliffe A, Tipu HN, Kuehn HS, Rosenzweig SD, Turro E, Tavaré S, Thrasher AJ, Jodrell DI, Smith KGC, Boztug K, Milner JD, Thaventhiran JED.

J Exp Med. 2019 Sep 2;216(9):1986-1998. doi: 10.1084/jem.20190344. Epub 2019 Jun 24.

16.

Safety and early efficacy outcomes for lentiviral fibroblast gene therapy in recessive dystrophic epidermolysis bullosa.

Lwin SM, Syed F, Di WL, Kadiyirire T, Liu L, Guy A, Petrova A, Abdul-Wahab A, Reid F, Phillips R, Elstad M, Georgiadis C, Aristodemou S, Lovell PA, McMillan JR, Mee J, Miskinyte S, Titeux M, Ozoemena L, Pramanik R, Serrano S, Rowles R, Maurin C, Orrin E, Martinez-Queipo M, Rashidghamat E, Tziotzios C, Onoufriadis A, Chen M, Chan L, Farzaneh F, Del Rio M, Tolar J, Bauer JW, Larcher F, Antoniou MN, Hovnanian A, Thrasher AJ, Mellerio JE, Qasim W, McGrath JA.

JCI Insight. 2019 Jun 6;4(11). pii: 126243. doi: 10.1172/jci.insight.126243. eCollection 2019 Jun 6.

17.

In Utero Transplantation of Expanded Autologous Amniotic Fluid Stem Cells Results in Long-Term Hematopoietic Engraftment.

Loukogeorgakis SP, Shangaris P, Bertin E, Franzin C, Piccoli M, Pozzobon M, Subramaniam S, Tedeschi A, Kim AG, Li H, Fachin CG, Dias AIBS, Stratigis JD, Ahn NJ, Thrasher AJ, Bonfanti P, Peranteau WH, David AL, Flake AW, De Coppi P.

Stem Cells. 2019 Sep;37(9):1176-1188. doi: 10.1002/stem.3039. Epub 2019 Jul 29.

18.

How I manage patients with Wiskott Aldrich syndrome.

Rivers E, Worth A, Thrasher AJ, Burns SO.

Br J Haematol. 2019 May;185(4):647-655. doi: 10.1111/bjh.15831. Epub 2019 Mar 12. Review.

PMID:
30864154
19.

Codon-Optimization of Wild-Type Adeno-Associated Virus Capsid Sequences Enhances DNA Family Shuffling while Conserving Functionality.

Cabanes-Creus M, Ginn SL, Amaya AK, Liao SHY, Westhaus A, Hallwirth CV, Wilmott P, Ward J, Dilworth KL, Santilli G, Rybicki A, Nakai H, Thrasher AJ, Filip AC, Alexander IE, Lisowski L.

Mol Ther Methods Clin Dev. 2018 Nov 1;12:71-84. doi: 10.1016/j.omtm.2018.10.016. eCollection 2019 Mar 15.

20.

Dendritic cell-expressed common gamma-chain recruits IL-15 for trans-presentation at the murine immunological synapse.

Beilin C, Choudhuri K, Bouma G, Malinova D, Llodra J, Stokes DL, Shimaoka M, Springer TA, Dustin ML, Thrasher AJ, Burns SO.

Version 2. Wellcome Open Res. 2018 Oct 17 [revised 2018 Jan 1];3:84. doi: 10.12688/wellcomeopenres.14493.2. eCollection 2018.

21.

EROS/CYBC1 mutations: Decreased NADPH oxidase function and chronic granulomatous disease.

Thomas DC, Charbonnier LM, Schejtman A, Aldhekri H, Coomber EL, Dufficy ER, Beenken AE, Lee JC, Clare S, Speak AO, Thrasher AJ, Santilli G, Al-Mousa H, Alkuraya FS, Chatila TA, Smith KGC.

J Allergy Clin Immunol. 2019 Feb;143(2):782-785.e1. doi: 10.1016/j.jaci.2018.09.019. Epub 2018 Oct 9. No abstract available.

22.

One hundred percent survival after transplantation of 34 patients with Wiskott-Aldrich syndrome over 20 years.

Elfeky RA, Furtado-Silva JM, Chiesa R, Rao K, Amrolia P, Lucchini G, Gilmour K, Adams S, Bibi S, Worth A, Thrasher AJ, Qasim W, Veys P.

J Allergy Clin Immunol. 2018 Nov;142(5):1654-1656.e7. doi: 10.1016/j.jaci.2018.06.042. Epub 2018 Jul 25. No abstract available.

PMID:
30055182
23.

Bleeding and splenectomy in Wiskott-Aldrich syndrome: A single-centre experience.

Rivers E, Worth A, Thrasher AJ, Burns SO.

J Allergy Clin Immunol Pract. 2019 Mar;7(3):1042-1044.e1. doi: 10.1016/j.jaip.2018.07.009. Epub 2018 Jul 23. No abstract available.

PMID:
30048768
24.

Age-Related Seroprevalence of Antibodies Against AAV-LK03 in a UK Population Cohort.

Perocheau DP, Cunningham S, Lee J, Antinao Diaz J, Waddington SN, Gilmour K, Eaglestone S, Lisowski L, Thrasher AJ, Alexander IE, Gissen P, Baruteau J.

Hum Gene Ther. 2019 Jan;30(1):79-87. doi: 10.1089/hum.2018.098. Epub 2018 Oct 19.

25.

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.

26.

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.

27.

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.

28.

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.

29.

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
30.

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.

31.

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.

32.

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 Oct;142(4):1285-1296. doi: 10.1016/j.jaci.2018.01.039. Epub 2018 Mar 2.

33.

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 Oct;142(4):1272-1284. doi: 10.1016/j.jaci.2017.12.1000. Epub 2018 Feb 6.

34.

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 Jul - Aug;6(4):1418-1420.e10. doi: 10.1016/j.jaip.2017.12.008. Epub 2018 Jan 20. Review. No abstract available.

35.

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.

36.

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.

37.

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
38.

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.

39.

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.

40.

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.

41.

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.

42.

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.

43.

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.

44.

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.

45.

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.

46.

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.

47.

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.

48.

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.

49.

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.

50.

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.

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