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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.


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.


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.


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.


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.


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.


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.


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.


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.


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.


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.


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.


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.


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.


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.


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.


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.


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.


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.


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.


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.


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.


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.


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.


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.


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.


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.


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.


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.


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.


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.


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.


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.


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.


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.


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.


Continuous local delivery of interferon-β stabilizes tumor vasculature in an orthotopic glioblastoma xenograft resection model.

Denbo JW, Williams RF, Orr WS, Sims TL, Ng CY, Zhou J, Spence Y, Morton CL, Nathwani AC, Duntsch C, Pfeffer LM, Davidoff AM.

Surgery. 2011 Sep;150(3):497-504. doi: 10.1016/j.surg.2011.07.044.


Successful attenuation of humoral immunity to viral capsid and transgenic protein following AAV-mediated gene transfer with a non-depleting CD4 antibody and cyclosporine.

McIntosh JH, Cochrane M, Cobbold S, Waldmann H, Nathwani SA, Davidoff AM, Nathwani AC.

Gene Ther. 2012 Jan;19(1):78-85. doi: 10.1038/gt.2011.64. Epub 2011 Jun 30.


Stable human FIX expression after 0.9G intrauterine gene transfer of self-complementary adeno-associated viral vector 5 and 8 in macaques.

Mattar CN, Nathwani AC, Waddington SN, Dighe N, Kaeppel C, Nowrouzi A, Mcintosh J, Johana NB, Ogden B, Fisk NM, Davidoff AM, David A, Peebles D, Valentine MB, Appelt JU, von Kalle C, Schmidt M, Biswas A, Choolani M, Chan JK.

Mol Ther. 2011 Nov;19(11):1950-60. doi: 10.1038/mt.2011.107. Epub 2011 May 31.


Good manufacturing practice production of self-complementary serotype 8 adeno-associated viral vector for a hemophilia B clinical trial.

Allay JA, Sleep S, Long S, Tillman DM, Clark R, Carney G, Fagone P, McIntosh JH, Nienhuis AW, Davidoff AM, Nathwani AC, Gray JT.

Hum Gene Ther. 2011 May;22(5):595-604. doi: 10.1089/hum.2010.202.


Long-term safety and efficacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins.

Nathwani AC, Rosales C, McIntosh J, Rastegarlari G, Nathwani D, Raj D, Nawathe S, Waddington SN, Bronson R, Jackson S, Donahue RE, High KA, Mingozzi F, Ng CY, Zhou J, Spence Y, McCarville MB, Valentine M, Allay J, Coleman J, Sleep S, Gray JT, Nienhuis AW, Davidoff AM.

Mol Ther. 2011 May;19(5):876-85. doi: 10.1038/mt.2010.274. Epub 2011 Jan 18.


Codon optimization of human factor VIII cDNAs leads to high-level expression.

Ward NJ, Buckley SM, Waddington SN, Vandendriessche T, Chuah MK, Nathwani AC, McIntosh J, Tuddenham EG, Kinnon C, Thrasher AJ, McVey JH.

Blood. 2011 Jan 20;117(3):798-807. doi: 10.1182/blood-2010-05-282707. Epub 2010 Nov 1.


Maturation of tumor vasculature by interferon-beta disrupts the vascular niche of glioma stem cells.

Williams RF, Sims TL, Tracey L, Myers AL, Ng CY, Poppleton H, Nathwani AC, Davidoff AM.

Anticancer Res. 2010 Sep;30(9):3301-8.


Recombinant adeno-associated virus-mediated in utero gene transfer gives therapeutic transgene expression in the sheep.

David AL, McIntosh J, Peebles DM, Cook T, Waddington S, Weisz B, Wigley V, Abi-Nader K, Boyd M, Davidoff AM, Nathwani AC.

Hum Gene Ther. 2011 Apr;22(4):419-26. doi: 10.1089/hum.2010.007. Epub 2011 Feb 2.


An introduction to induced pluripotent stem cells.

Hanley J, Rastegarlari G, Nathwani AC.

Br J Haematol. 2010 Oct;151(1):16-24. doi: 10.1111/j.1365-2141.2010.08296.x. Epub 2010 Jul 27. Review.


Targeting multiple angiogenic pathways for the treatment of neuroblastoma.

Williams RF, Myers AL, Sims TL, Ng CY, Nathwani AC, Davidoff AM.

J Pediatr Surg. 2010 Jun;45(6):1103-9. doi: 10.1016/j.jpedsurg.2010.02.073.


Improved intratumoral oxygenation through vascular normalization increases glioma sensitivity to ionizing radiation.

McGee MC, Hamner JB, Williams RF, Rosati SF, Sims TL, Ng CY, Gaber MW, Calabrese C, Wu J, Nathwani AC, Duntsch C, Merchant TE, Davidoff AM.

Int J Radiat Oncol Biol Phys. 2010 Apr;76(5):1537-45. doi: 10.1016/j.ijrobp.2009.12.010.


IFN-beta restricts tumor growth and sensitizes alveolar rhabdomyosarcoma to ionizing radiation.

Sims TL, McGee M, Williams RF, Myers AL, Tracey L, Hamner JB, Ng C, Wu J, Gaber MW, McCarville B, Nathwani AC, Davidoff AM.

Mol Cancer Ther. 2010 Mar;9(3):761-71. doi: 10.1158/1535-7163.MCT-09-0800. Epub 2010 Mar 2.


NF-kappaB activation mediates resistance to IFN beta in MLL-rearranged acute lymphoblastic leukemia.

Tracey L, Streck CJ, Du Z, Williams RF, Pfeffer LM, Nathwani AC, Davidoff AM.

Leukemia. 2010 Apr;24(4):806-12. doi: 10.1038/leu.2010.2. Epub 2010 Feb 4.


AAV-mediated knockdown of peripherin-2 in vivo using miRNA-based hairpins.

Georgiadis A, Tschernutter M, Bainbridge JW, Robbie SJ, McIntosh J, Nathwani AC, Smith AJ, Ali RR.

Gene Ther. 2010 Apr;17(4):486-93. doi: 10.1038/gt.2009.162. Epub 2009 Dec 10.


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