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Items: 1 to 50 of 131

1.

Ibrutinib enhances the efficacy of ROR1 bispecific T cell engager mediated cytotoxicity in chronic lymphocytic leukaemia.

Gohil SH, Evans R, Harasser M, El-Kholy M, Paredes-Moscosso SR, Della Peruta M, Nathwani AC.

Br J Haematol. 2019 Jul;186(2):380-382. doi: 10.1111/bjh.15911. Epub 2019 Apr 7. No abstract available. Erratum in: Br J Haematol. 2019 Sep;186(6):908.

PMID:
30957227
2.

Intergenerational transmission of child maltreatment: Predictors of child emotional maltreatment among 11 to 17 years old children residing in communities of Karachi, Pakistan.

Lakhdir MPA, Nathwani AA, Ali NA, Farooq S, Azam SI, Khaliq A, Kadir MM.

Child Abuse Negl. 2019 May;91:109-115. doi: 10.1016/j.chiabu.2019.03.004. Epub 2019 Mar 8.

PMID:
30856598
3.

Potential limits of AAV-based gene therapy with the use of new transgenes expressing factor IX fusion proteins.

Le Quellec S, Dane A, Enjolras N, McIntosh J, Rosales C, Negrier C, Nathwani A.

Haemophilia. 2019 Jan;25(1):e11-e18. doi: 10.1111/hae.13651. Epub 2018 Dec 6.

PMID:
30520547
4.

Therapeutic expression of human clotting factors IX and X following adeno-associated viral vector-mediated intrauterine gene transfer in early-gestation fetal macaques.

Chan JKY, Gil-Farina I, Johana N, Rosales C, Tan YW, Ceiler J, Mcintosh J, Ogden B, Waddington SN, Schmidt M, Biswas A, Choolani M, Nathwani AC, Mattar CNZ.

FASEB J. 2019 Mar;33(3):3954-3967. doi: 10.1096/fj.201801391R. Epub 2018 Dec 5.

5.

Immunosuppression overcomes insulin- and vector-specific immune responses that limit efficacy of AAV2/8-mediated insulin gene therapy in NOD mice.

Recino A, Gan SU, Sia KC, Sawyer Y, Trendell J, Kay R, Gribble FM, Reimann F, Foale R, Notaridou M, Holmes N, Lever A, Lee KO, Nathwani A, Cooke A, Calne R, Wallberg M.

Gene Ther. 2019 Feb;26(1-2):40-56. doi: 10.1038/s41434-018-0052-5. Epub 2018 Dec 4.

PMID:
30514969
6.

Mbd3 Promotes Reprogramming of Primary Human Fibroblasts.

Jaffer S, Goh P, Abbasian M, Nathwani AC.

Int J Stem Cells. 2018 Nov 30;11(2):235-241. doi: 10.15283/ijsc18036.

7.

The murine lung as a factory to produce secreted intrapulmonary and circulatory proteins.

Paul-Smith MC, Pytel KM, Gelinas JF, McIntosh J, Pringle I, Davies L, Chan M, Meng C, Bell R, Cammack L, Moran C, Cameron L, Inoue M, Tsugumine S, Hironaka T, Gill DR, Hyde SC, Nathwani A, Alton EWFW, Griesenbach U.

Gene Ther. 2018 Aug;25(5):345-358. doi: 10.1038/s41434-018-0025-8. Epub 2018 Jul 18.

8.

Recent advances in developing specific therapies for haemophilia.

Ling G, Nathwani AC, Tuddenham EGD.

Br J Haematol. 2018 Apr;181(2):161-172. doi: 10.1111/bjh.15084. Epub 2018 Jan 23. Review.

PMID:
29359795
9.

LenD: a study to establish the safety and efficacy of lenalidomide and dexamethasone in patients with relapsed or refractory chronic lymphocytic leukemia.

Gohil SH, Maciocia N, Patrick P, Roberts T, Counsell N, Smith P, Clifton-Hadley L, Cwynarski K, Pettitt A, Nathwani AC.

Leuk Lymphoma. 2018 Jul;59(7):1730-1733. doi: 10.1080/10428194.2017.1399311. Epub 2017 Nov 22. No abstract available.

PMID:
29164978
10.

Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity.

Paulk NK, Pekrun K, Zhu E, Nygaard S, Li B, Xu J, Chu K, Leborgne C, Dane AP, Haft A, Zhang Y, Zhang F, Morton C, Valentine MB, Davidoff AM, Nathwani AC, Mingozzi F, Grompe M, Alexander IE, Lisowski L, Kay MA.

Mol Ther. 2018 Jan 3;26(1):289-303. doi: 10.1016/j.ymthe.2017.09.021. Epub 2017 Sep 25.

11.

Gene Therapy for Hemophilia.

Nathwani AC, Davidoff AM, Tuddenham EGD.

Hematol Oncol Clin North Am. 2017 Oct;31(5):853-868. doi: 10.1016/j.hoc.2017.06.011. Review.

PMID:
28895852
12.

Advances in Gene Therapy for Hemophilia.

Nathwani AC, Davidoff AM, Tuddenham EGD.

Hum Gene Ther. 2017 Nov;28(11):1004-1012. doi: 10.1089/hum.2017.167. Review.

PMID:
28835123
13.

An ROR1 bi-specific T-cell engager provides effective targeting and cytotoxicity against a range of solid tumors.

Gohil SH, Paredes-Moscosso SR, Harrasser M, Vezzalini M, Scarpa A, Morris E, Davidoff AM, Sorio C, Nathwani AC, Della Peruta M.

Oncoimmunology. 2017 May 17;6(7):e1326437. doi: 10.1080/2162402X.2017.1326437. eCollection 2017.

14.

Targeting Histone Demethylases in MYC-Driven Neuroblastomas with Ciclopirox.

Yang J, Milasta S, Hu D, AlTahan AM, Interiano RB, Zhou J, Davidson J, Low J, Lin W, Bao J, Goh P, Nathwani AC, Wang R, Wang Y, Ong SS, Boyd VA, Young B, Das S, Shelat A, Wu Y, Li Z, Zheng JJ, Mishra A, Cheng Y, Qu C, Peng J, Green DR, White S, Guy RK, Chen T, Davidoff AM.

Cancer Res. 2017 Sep 1;77(17):4626-4638. doi: 10.1158/0008-5472.CAN-16-0826. Epub 2017 Jul 6.

15.

The Transcription Factor ZNF683/HOBIT Regulates Human NK-Cell Development.

Post M, Cuapio A, Osl M, Lehmann D, Resch U, Davies DM, Bilban M, Schlechta B, Eppel W, Nathwani A, Stoiber D, Spanholtz J, Casanova E, Hofer E.

Front Immunol. 2017 May 15;8:535. doi: 10.3389/fimmu.2017.00535. eCollection 2017.

16.

In Utero Transfer of Adeno-Associated Viral Vectors Produces Long-Term Factor IX Levels in a Cynomolgus Macaque Model.

Mattar CNZ, Gil-Farina I, Rosales C, Johana N, Tan YYW, McIntosh J, Kaeppel C, Waddington SN, Biswas A, Choolani M, Schmidt M, Nathwani AC, Chan JKY.

Mol Ther. 2017 Aug 2;25(8):1843-1853. doi: 10.1016/j.ymthe.2017.04.003. Epub 2017 Apr 24.

17.

Gene Therapy for Hemophilia.

Nienhuis AW, Nathwani AC, Davidoff AM.

Mol Ther. 2017 May 3;25(5):1163-1167. doi: 10.1016/j.ymthe.2017.03.033. Epub 2017 Apr 11. Review.

18.

Polonium-210 poisoning: a first-hand account.

Nathwani AC, Down JF, Goldstone J, Yassin J, Dargan PI, Virchis A, Gent N, Lloyd D, Harrison JD.

Lancet. 2016 Sep 10;388(10049):1075-1080. doi: 10.1016/S0140-6736(16)00144-6. Epub 2016 Jul 23.

PMID:
27461439
19.

A high-throughput sequencing test for diagnosing inherited bleeding, thrombotic, and platelet disorders.

Simeoni I, Stephens JC, Hu F, Deevi SV, Megy K, Bariana TK, Lentaigne C, Schulman S, Sivapalaratnam S, Vries MJ, Westbury SK, Greene D, Papadia S, Alessi MC, Attwood AP, Ballmaier M, Baynam G, Bermejo E, Bertoli M, Bray PF, Bury L, Cattaneo M, Collins P, Daugherty LC, Favier R, French DL, Furie B, Gattens M, Germeshausen M, Ghevaert C, Goodeve AC, Guerrero JA, Hampshire DJ, Hart DP, Heemskerk JW, Henskens YM, Hill M, Hogg N, Jolley JD, Kahr WH, Kelly AM, Kerr R, Kostadima M, Kunishima S, Lambert MP, Liesner R, López JA, Mapeta RP, Mathias M, Millar CM, Nathwani A, Neerman-Arbez M, Nurden AT, Nurden P, Othman M, Peerlinck K, Perry DJ, Poudel P, Reitsma P, Rondina MT, Smethurst PA, Stevenson W, Szkotak A, Tuna S, van Geet C, Whitehorn D, Wilcox DA, Zhang B, Revel-Vilk S, Gresele P, Bellissimo DB, Penkett CJ, Laffan MA, Mumford AD, Rendon A, Gomez K, Freson K, Ouwehand WH, Turro E.

Blood. 2016 Jun 9;127(23):2791-803. doi: 10.1182/blood-2015-12-688267. Epub 2016 Apr 15.

20.

Distribution of AAV8 particles in cell lysates and culture media changes with time and is dependent on the recombinant vector.

Piras BA, Drury JE, Morton CL, Spence Y, Lockey TD, Nathwani AC, Davidoff AM, Meagher MM.

Mol Ther Methods Clin Dev. 2016 Mar 30;3:16015. doi: 10.1038/mtm.2016.15. eCollection 2016.

21.

Genetic Targeting of the Albumin Locus to Treat Hemophilia.

Davidoff AM, Nathwani AC.

N Engl J Med. 2016 Mar 31;374(13):1288-90. doi: 10.1056/NEJMcibr1600347. No abstract available.

PMID:
27028920
22.

Gene Therapy for Hemophilia.

Nienhuis AW, Nathwani AC, Davidoff AM.

Hum Gene Ther. 2016 Apr;27(4):305-8. doi: 10.1089/hum.2016.018. Review.

PMID:
27021603
23.

Correction of Murine Diabetic Hyperglycaemia With A Single Systemic Administration of An AAV2/8 Vector Containing A Novel Codon Optimized Human Insulin Gene.

Gan SU, Notaridou M, Fu ZY, Lee KO, Sia KC, Nathwani AC, Della Peruta M, Calne RY.

Curr Gene Ther. 2016;16(1):65-72.

PMID:
26795016
24.

New and improved AAVenues: current status of hemophilia B gene therapy.

Brimble MA, Reiss UM, Nathwani AC, Davidoff AM.

Expert Opin Biol Ther. 2016;16(1):79-92. doi: 10.1517/14712598.2015.1106475. Epub 2015 Nov 2. Review.

PMID:
26524468
25.

Haemophilia gene therapy: Progress and challenges.

Lheriteau E, Davidoff AM, Nathwani AC.

Blood Rev. 2015 Sep;29(5):321-8. doi: 10.1016/j.blre.2015.03.002. Epub 2015 Mar 26. Review.

PMID:
26049173
26.

The role of histone demethylase KDM4B in Myc signaling in neuroblastoma.

Yang J, AlTahan AM, Hu D, Wang Y, Cheng PH, Morton CL, Qu C, Nathwani AC, Shohet JM, Fotsis T, Koster J, Versteeg R, Okada H, Harris AL, Davidoff AM.

J Natl Cancer Inst. 2015 Apr 29;107(6):djv080. doi: 10.1093/jnci/djv080. Print 2015 Jun.

27.

Generation of Integration-Free Patient Specific iPS Cells Using Episomal Plasmids Under Feeder Free Conditions.

Caxaria S, Arthold S, Nathwani AC, Goh PA.

Methods Mol Biol. 2016;1353:355-66. doi: 10.1007/7651_2015_204.

PMID:
25701132
28.

Minimal residual disease detection with tumor-specific CD160 correlates with event-free survival in chronic lymphocytic leukemia.

Farren TW, Giustiniani J, Fanous M, Liu F, Macey MG, Wright F, Prentice A, Nathwani A, Agrawal SG.

Blood Cancer J. 2015 Jan 23;5:e273. doi: 10.1038/bcj.2014.92.

29.

Rewiring of sIgM-Mediated Intracellular Signaling through the CD180 Toll-like Receptor.

Porakishvili N, Vispute K, Steele AJ, Rajakaruna N, Kulikova N, Tsertsvadze T, Nathwani A, Damle RN, Clark EA, Rai KR, Chiorazzi N, Lydyard PM.

Mol Med. 2015 Apr;21(1):46-57. doi: 10.2119/molmed.2014.00265. Epub 2015 Jan 14.

30.

Preferential targeting of disseminated liver tumors using a recombinant adeno-associated viral vector.

Della Peruta M, Badar A, Rosales C, Chokshi S, Kia A, Nathwani D, Galante E, Yan R, Arstad E, Davidoff AM, Williams R, Lythgoe MF, Nathwani AC.

Hum Gene Ther. 2015 Feb;26(2):94-103. doi: 10.1089/hum.2014.052.

31.

Long-term safety and efficacy of factor IX gene therapy in hemophilia B.

Nathwani AC, Reiss UM, Tuddenham EG, Rosales C, Chowdary P, McIntosh J, Della Peruta M, Lheriteau E, Patel N, Raj D, Riddell A, Pie J, Rangarajan S, Bevan D, Recht M, Shen YM, Halka KG, Basner-Tschakarjan E, Mingozzi F, High KA, Allay J, Kay MA, Ng CY, Zhou J, Cancio M, Morton CL, Gray JT, Srivastava D, Nienhuis AW, Davidoff AM.

N Engl J Med. 2014 Nov 20;371(21):1994-2004. doi: 10.1056/NEJMoa1407309.

32.

Our journey to successful gene therapy for hemophilia B.

Nathwani AC, Nienhuis AW, Davidoff AM.

Hum Gene Ther. 2014 Nov;25(11):923-6. doi: 10.1089/hum.2014.2540. No abstract available.

33.

Human induced pluripotent stem cell derived erythroblasts can undergo definitive erythropoiesis and co-express gamma and beta globins.

Yang CT, French A, Goh PA, Pagnamenta A, Mettananda S, Taylor J, Knight S, Nathwani A, Roberts DJ, Watt SM, Carpenter L.

Br J Haematol. 2014 Aug;166(3):435-48. doi: 10.1111/bjh.12910. Epub 2014 May 16.

34.

Current status of haemophilia gene therapy.

High KH, Nathwani A, Spencer T, Lillicrap D.

Haemophilia. 2014 May;20 Suppl 4:43-9. doi: 10.1111/hae.12411. Review.

PMID:
24762274
35.

Rituximab plus chlorambucil as first-line treatment for chronic lymphocytic leukemia: Final analysis of an open-label phase II study.

Hillmen P, Gribben JG, Follows GA, Milligan D, Sayala HA, Moreton P, Oscier DG, Dearden CE, Kennedy DB, Pettitt AR, Nathwani A, Varghese A, Cohen D, Rawstron A, Oertel S, Pocock CF.

J Clin Oncol. 2014 Apr 20;32(12):1236-41. doi: 10.1200/JCO.2013.49.6547. Epub 2014 Mar 17.

36.

Progress towards gene therapy for haemophilia B.

Patel N, Reiss U, Davidoff AM, Nathwani AC.

Int J Hematol. 2014 Apr;99(4):372-6. doi: 10.1007/s12185-014-1523-0. Epub 2014 Feb 6. Review.

37.

A systematic evaluation of integration free reprogramming methods for deriving clinically relevant patient specific induced pluripotent stem (iPS) cells.

Goh PA, Caxaria S, Casper C, Rosales C, Warner TT, Coffey PJ, Nathwani AC.

PLoS One. 2013 Nov 26;8(11):e81622. doi: 10.1371/journal.pone.0081622. eCollection 2013.

38.

Developments in the treatment of hemophilia B: focus on emerging gene therapy.

Cancio MI, Reiss UM, Nathwani AC, Davidoff AM, Gray JT.

Appl Clin Genet. 2013 Oct 18;6:91-101. doi: 10.2147/TACG.S31928. Review.

39.

Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant.

McIntosh J, Lenting PJ, Rosales C, Lee D, Rabbanian S, Raj D, Patel N, Tuddenham EG, Christophe OD, McVey JH, Waddington S, Nienhuis AW, Gray JT, Fagone P, Mingozzi F, Zhou SZ, High KA, Cancio M, Ng CY, Zhou J, Morton CL, Davidoff AM, Nathwani AC.

Blood. 2013 Apr 25;121(17):3335-44. doi: 10.1182/blood-2012-10-462200. Epub 2013 Feb 20.

40.

Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases.

Tremblay JP, Xiao X, Aartsma-Rus A, Barbas C, Blau HM, Bogdanove AJ, Boycott K, Braun S, Breakefield XO, Bueren JA, Buschmann M, Byrne BJ, Calos M, Cathomen T, Chamberlain J, Chuah M, Cornetta K, Davies KE, Dickson JG, Duchateau P, Flotte TR, Gaudet D, Gersbach CA, Gilbert R, Glorioso J, Herzog RW, High KA, Huang W, Huard J, Joung JK, Liu D, Liu D, Lochmüller H, Lustig L, Martens J, Massie B, Mavilio F, Mendell JR, Nathwani A, Ponder K, Porteus M, Puymirat J, Samulski J, Takeda S, Thrasher A, VandenDriessche T, Wei Y, Wilson JM, Wilton SD, Wolfe JH, Gao G.

Mol Ther. 2013 Feb;21(2):266-8. doi: 10.1038/mt.2013.4. No abstract available.

41.

The case for intrauterine gene therapy.

Mattar CN, Waddington SN, Biswas A, Davidoff AM, Choolani M, Chan JK, Nathwani AC.

Best Pract Res Clin Obstet Gynaecol. 2012 Oct;26(5):697-709. doi: 10.1016/j.bpobgyn.2012.06.006. Epub 2012 Jul 20. Review.

PMID:
22819290
42.

Transient B cell depletion or improved transgene expression by codon optimization promote tolerance to factor VIII in gene therapy.

Sack BK, Merchant S, Markusic DM, Nathwani AC, Davidoff AM, Byrne BJ, Herzog RW.

PLoS One. 2012;7(5):e37671. doi: 10.1371/journal.pone.0037671. Epub 2012 May 24.

43.

Vector systems for prenatal gene therapy: principles of adeno-associated virus vector design and production.

Binny CJ, Nathwani AC.

Methods Mol Biol. 2012;891:109-31. doi: 10.1007/978-1-61779-873-3_6.

PMID:
22648770
44.

Systemic errors in quantitative polymerase chain reaction titration of self-complementary adeno-associated viral vectors and improved alternative methods.

Fagone P, Wright JF, Nathwani AC, Nienhuis AW, Davidoff AM, Gray JT.

Hum Gene Ther Methods. 2012 Feb;23(1):1-7. doi: 10.1089/hgtb.2011.104.

45.

Comparison of cryoprecipitate with fibrinogen concentrate for acquired hypofibrinogenaemia.

Theodoulou A, Berryman J, Nathwani A, Scully M.

Transfus Apher Sci. 2012 Apr;46(2):159-62. doi: 10.1016/j.transci.2011.11.005. Epub 2012 Jan 9.

PMID:
22227402
46.

Adenovirus-associated virus vector-mediated gene transfer in hemophilia B.

Nathwani AC, Tuddenham EG, Rangarajan S, Rosales C, McIntosh J, Linch DC, Chowdary P, Riddell A, Pie AJ, Harrington C, O'Beirne J, Smith K, Pasi J, Glader B, Rustagi P, Ng CY, Kay MA, Zhou J, Spence Y, Morton CL, Allay J, Coleman J, Sleep S, Cunningham JM, Srivastava D, Basner-Tschakarjan E, Mingozzi F, High KA, Gray JT, Reiss UM, Nienhuis AW, Davidoff AM.

N Engl J Med. 2011 Dec 22;365(25):2357-65. doi: 10.1056/NEJMoa1108046. Epub 2011 Dec 10.

47.

AAV-mediated gene transfer in the perinatal period results in expression of FVII at levels that protect against fatal spontaneous hemorrhage.

Binny C, McIntosh J, Della Peruta M, Kymalainen H, Tuddenham EG, Buckley SM, Waddington SN, McVey JH, Spence Y, Morton CL, Thrasher AJ, Gray JT, Castellino FJ, Tarantal AF, Davidoff AM, Nathwani AC.

Blood. 2012 Jan 26;119(4):957-66. doi: 10.1182/blood-2011-09-377630. Epub 2011 Dec 1.

48.

In utero administration of Ad5 and AAV pseudotypes to the fetal brain leads to efficient, widespread and long-term gene expression.

Rahim AA, Wong AM, Ahmadi S, Hoefer K, Buckley SM, Hughes DA, Nathwani AN, Baker AH, McVey JH, Cooper JD, Waddington SN.

Gene Ther. 2012 Sep;19(9):936-46. doi: 10.1038/gt.2011.157. Epub 2011 Nov 10.

PMID:
22071970
49.

AAV vector biology in primates: finding the missing link?

Herzog RW, Davidoff AM, Markusic DM, Nathwani AC.

Mol Ther. 2011 Nov;19(11):1923-4. doi: 10.1038/mt.2011.218. No abstract available.

50.

Self-complementary adeno-associated viral vectors for gene therapy of hemophilia B: progress and challenges.

Raj D, Davidoff AM, Nathwani AC.

Expert Rev Hematol. 2011 Oct;4(5):539-49. doi: 10.1586/ehm.11.48. Review.

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