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Items: 1 to 50 of 55

1.

The Landscape of Early Clinical Gene Therapies outside of Oncology.

Rittié L, Athanasopoulos T, Calero-Garcia M, Davies ML, Dow DJ, Howe SJ, Morrison A, Ricciardelli I, Saudemont A, Jespers L, Clay TM.

Mol Ther. 2019 Oct 2;27(10):1706-1717. doi: 10.1016/j.ymthe.2019.09.002. Epub 2019 Sep 6. Review.

PMID:
31526597
2.

Inhibition of Mitochondrial Complex I Impairs Release of α-Galactosidase by Jurkat Cells.

Lambert JRA, Howe SJ, Rahim AA, Burke DG, Heales SJR.

Int J Mol Sci. 2019 Sep 5;20(18). pii: E4349. doi: 10.3390/ijms20184349.

3.

The myotonic dystrophy experience: a North American cross-sectional study.

Hagerman KA, Howe SJ, Heatwole CR; Christopher Project Reference Group.

Muscle Nerve. 2019 Apr;59(4):457-464. doi: 10.1002/mus.26420. Epub 2019 Feb 5.

4.

Argininosuccinic aciduria fosters neuronal nitrosative stress reversed by Asl gene transfer.

Baruteau J, Perocheau DP, Hanley J, Lorvellec M, Rocha-Ferreira E, Karda R, Ng J, Suff N, Diaz JA, Rahim AA, Hughes MP, Banushi B, Prunty H, Hristova M, Ridout DA, Virasami A, Heales S, Howe SJ, Buckley SMK, Mills PB, Gissen P, Waddington SN.

Nat Commun. 2018 Aug 29;9(1):3505. doi: 10.1038/s41467-018-05972-1.

5.

Foamy Virus Vectors Transduce Visceral Organs and Hippocampal Structures following In Vivo Delivery to Neonatal Mice.

Counsell JR, Karda R, Diaz JA, Carey L, Wiktorowicz T, Buckley SMK, Ameri S, Ng J, Baruteau J, Almeida F, de Silva R, Simone R, Lugarà E, Lignani G, Lindemann D, Rethwilm A, Rahim AA, Waddington SN, Howe SJ.

Mol Ther Nucleic Acids. 2018 Sep 7;12:626-634. doi: 10.1016/j.omtn.2018.07.006. Epub 2018 Aug 3.

6.

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.

7.

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.

8.

Enhancement of mouse hematopoietic stem/progenitor cell function via transient gene delivery using integration-deficient lentiviral vectors.

Alonso-Ferrero ME, van Til NP, Bartolovic K, Mata MF, Wagemaker G, Moulding D, Williams DA, Kinnon C, Waddington SN, Milsom MD, Howe SJ.

Exp Hematol. 2018 Jan;57:21-29. doi: 10.1016/j.exphem.2017.09.003. Epub 2017 Sep 11.

9.

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.

10.

Eliminating HIV-1 Packaging Sequences from Lentiviral Vector Proviruses Enhances Safety and Expedites Gene Transfer for Gene Therapy.

Vink CA, Counsell JR, Perocheau DP, Karda R, Buckley SMK, Brugman MH, Galla M, Schambach A, McKay TR, Waddington SN, Howe SJ.

Mol Ther. 2017 Aug 2;25(8):1790-1804. doi: 10.1016/j.ymthe.2017.04.028. Epub 2017 May 24.

11.

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.

12.

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.

13.

BMI-1 extends proliferative potential of human bronchial epithelial cells while retaining their mucociliary differentiation capacity.

Munye MM, Shoemark A, Hirst RA, Delhove JM, Sharp TV, McKay TR, O'Callaghan C, Baines DL, Howe SJ, Hart SL.

Am J Physiol Lung Cell Mol Physiol. 2017 Feb 1;312(2):L258-L267. doi: 10.1152/ajplung.00471.2016. Epub 2016 Dec 15.

14.

Regulation of post-Golgi LH3 trafficking is essential for collagen homeostasis.

Banushi B, Forneris F, Straatman-Iwanowska A, Strange A, Lyne AM, Rogerson C, Burden JJ, Heywood WE, Hanley J, Doykov I, Straatman KR, Smith H, Bem D, Kriston-Vizi J, Ariceta G, Risteli M, Wang C, Ardill RE, Zaniew M, Latka-Grot J, Waddington SN, Howe SJ, Ferraro F, Gjinovci A, Lawrence S, Marsh M, Girolami M, Bozec L, Mills K, Gissen P.

Nat Commun. 2016 Jul 20;7:12111. doi: 10.1038/ncomms12111.

15.

Minicircle DNA Provides Enhanced and Prolonged Transgene Expression Following Airway Gene Transfer.

Munye MM, Tagalakis AD, Barnes JL, Brown RE, McAnulty RJ, Howe SJ, Hart SL.

Sci Rep. 2016 Mar 15;6:23125. doi: 10.1038/srep23125.

16.

In vivo bioimaging with tissue-specific transcription factor activated luciferase reporters.

Buckley SM, Delhove JM, Perocheau DP, Karda R, Rahim AA, Howe SJ, Ward NJ, Birrell MA, Belvisi MG, Arbuthnot P, Johnson MR, Waddington SN, McKay TR.

Sci Rep. 2015 Jul 3;5:11842. doi: 10.1038/srep11842.

17.

Systemic gene delivery following intravenous administration of AAV9 to fetal and neonatal mice and late-gestation nonhuman primates.

Mattar CN, Wong AM, Hoefer K, Alonso-Ferrero ME, Buckley SM, Howe SJ, Cooper JD, Waddington SN, Chan JK, Rahim AA.

FASEB J. 2015 Sep;29(9):3876-88. doi: 10.1096/fj.14-269092. Epub 2015 Jun 10.

18.

Evidence for contribution of CD4+ CD25+ regulatory T cells in maintaining immune tolerance to human factor IX following perinatal adenovirus vector delivery.

Nivsarkar MS, Buckley SM, Parker AL, Perocheau D, McKay TR, Rahim AA, Howe SJ, Waddington SN.

J Immunol Res. 2015;2015:397879. doi: 10.1155/2015/397879. Epub 2015 Jan 31.

19.

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.

20.

Lentiviral labeling of mouse and human enteric nervous system stem cells for regenerative medicine studies.

Natarajan D, Cooper J, Choudhury S, Delalande JM, McCann C, Howe SJ, Thapar N, Burns AJ.

Neurogastroenterol Motil. 2014 Oct;26(10):1513-8. doi: 10.1111/nmo.12420. Epub 2014 Sep 8.

21.

Targeted expression of human folylpolyglutamate synthase for selective enhancement of methotrexate chemotherapy in osteosarcoma cells.

Bienemann K, Staege MS, Howe SJ, Sena-Esteves M, Hanenberg H, Kramm CM.

Cancer Gene Ther. 2013 Sep;20(9):514-20. doi: 10.1038/cgt.2013.48. Epub 2013 Aug 16.

PMID:
23949282
22.

Vector systems for prenatal gene therapy: principles of retrovirus vector design and production.

Howe SJ, Chandrashekran A.

Methods Mol Biol. 2012;891:85-107. doi: 10.1007/978-1-61779-873-3_5.

PMID:
22648769
23.

Hematopoietic stem cell and gene therapy corrects primary neuropathology and behavior in mucopolysaccharidosis IIIA mice.

Langford-Smith A, Wilkinson FL, Langford-Smith KJ, Holley RJ, Sergijenko A, Howe SJ, Bennett WR, Jones SA, Wraith J, Merry CL, Wynn RF, Bigger BW.

Mol Ther. 2012 Aug;20(8):1610-21. doi: 10.1038/mt.2012.82. Epub 2012 May 1.

24.

A small molecule modulator of prion protein increases human mesenchymal stem cell lifespan, ex vivo expansion, and engraftment to bone marrow in NOD/SCID mice.

Mohanty ST, Cairney CJ, Chantry AD, Madan S, Fernandes JA, Howe SJ, Moore HD, Thompson MJ, Chen B, Thrasher A, Keith WN, Bellantuono I.

Stem Cells. 2012 Jun;30(6):1134-43. doi: 10.1002/stem.1065.

25.

Retrovirus and lentivirus vector design and methods of cell conditioning.

Cooray S, Howe SJ, Thrasher AJ.

Methods Enzymol. 2012;507:29-57. doi: 10.1016/B978-0-12-386509-0.00003-X.

PMID:
22365768
26.

Long-term persistence of a polyclonal T cell repertoire after gene therapy for X-linked severe combined immunodeficiency.

Gaspar HB, Cooray S, Gilmour KC, Parsley KL, Adams S, Howe SJ, Al Ghonaium A, Bayford J, Brown L, Davies EG, Kinnon C, Thrasher AJ.

Sci Transl Med. 2011 Aug 24;3(97):97ra79. doi: 10.1126/scitranslmed.3002715.

27.

Insertion sites in engrafted cells cluster within a limited repertoire of genomic areas after gammaretroviral vector gene therapy.

Deichmann A, Brugman MH, Bartholomae CC, Schwarzwaelder K, Verstegen MM, Howe SJ, Arens A, Ott MG, Hoelzer D, Seger R, Grez M, Hacein-Bey-Abina S, Cavazzana-Calvo M, Fischer A, Paruzynski A, Gabriel R, Glimm H, Abel U, Cattoglio C, Mavilio F, Cassani B, Aiuti A, Dunbar CE, Baum C, Gaspar HB, Thrasher AJ, von Kalle C, Schmidt M, Wagemaker G.

Mol Ther. 2011 Nov;19(11):2031-9. doi: 10.1038/mt.2011.178. Epub 2011 Aug 23.

28.

Perinatal gene transfer to the liver.

McKay TR, Rahim AA, Buckley SM, Ward NJ, Chan JK, Howe SJ, Waddington SN.

Curr Pharm Des. 2011;17(24):2528-41. Review.

29.

Lentiviral vector integration profiles differ in rodent postmitotic tissues.

Bartholomae CC, Arens A, Balaggan KS, Yáñez-Muñoz RJ, Montini E, Howe SJ, Paruzynski A, Korn B, Appelt JU, Macneil A, Cesana D, Abel U, Glimm H, Naldini L, Ali RR, Thrasher AJ, von Kalle C, Schmidt M.

Mol Ther. 2011 Apr;19(4):703-10. doi: 10.1038/mt.2011.19. Epub 2011 Mar 1.

30.

Development of S/MAR minicircles for enhanced and persistent transgene expression in the mouse liver.

Argyros O, Wong SP, Fedonidis C, Tolmachov O, Waddington SN, Howe SJ, Niceta M, Coutelle C, Harbottle RP.

J Mol Med (Berl). 2011 May;89(5):515-29. doi: 10.1007/s00109-010-0713-3. Epub 2011 Feb 8.

PMID:
21301798
31.

Systemic gene transfer of polyethylenimine (PEI)-plasmid DNA complexes to neonatal mice.

Wong SP, Argyros O, Howe SJ, Harbottle RP.

J Control Release. 2011 Mar 30;150(3):298-306. doi: 10.1016/j.jconrel.2010.12.010. Epub 2010 Dec 28.

PMID:
21192993
32.

Correction of SCID-X1 using an enhancerless Vav promoter.

Almarza E, Zhang F, Santilli G, Blundell MP, Howe SJ, Thornhill SI, Bueren JA, Thrasher AJ.

Hum Gene Ther. 2011 Mar;22(3):263-70. doi: 10.1089/hum.2010.119. Epub 2011 Feb 7.

PMID:
20887212
33.

Gene delivery of a mutant TGFβ3 reduces markers of scar tissue formation after cutaneous wounding.

Waddington SN, Crossley R, Sheard V, Howe SJ, Buckley SM, Coughlan L, Gilham DE, Hawkins RE, McKay TR.

Mol Ther. 2010 Dec;18(12):2104-11. doi: 10.1038/mt.2010.174. Epub 2010 Aug 24.

34.

The differentiation and engraftment potential of mouse hematopoietic stem cells is maintained after bio-electrospray.

Bartolovic K, Mongkoldhumrongkul N, Waddington SN, Jayasinghe SN, Howe SJ.

Analyst. 2010 Jan;135(1):157-64. doi: 10.1039/b917813a. Epub 2009 Nov 13.

PMID:
20024196
35.

Comprehensive genomic access to vector integration in clinical gene therapy.

Gabriel R, Eckenberg R, Paruzynski A, Bartholomae CC, Nowrouzi A, Arens A, Howe SJ, Recchia A, Cattoglio C, Wang W, Faber K, Schwarzwaelder K, Kirsten R, Deichmann A, Ball CR, Balaggan KS, Yáñez-Muñoz RJ, Ali RR, Gaspar HB, Biasco L, Aiuti A, Cesana D, Montini E, Naldini L, Cohen-Haguenauer O, Mavilio F, Thrasher AJ, Glimm H, von Kalle C, Saurin W, Schmidt M.

Nat Med. 2009 Dec;15(12):1431-6. doi: 10.1038/nm.2057. Epub 2009 Nov 22.

PMID:
19966782
36.

Migratory and antigen presentation functions of IFN-producing killer dendritic cells.

Himoudi N, Yan M, Bouma G, Morgenstern D, Wallace R, Seddon B, Buddle J, Eddaoudi A, Howe SJ, Cooper N, Anderson J.

Cancer Res. 2009 Aug 15;69(16):6598-606. doi: 10.1158/0008-5472.CAN-09-0501. Epub 2009 Aug 4.

37.

Tolerance induction using lentiviral gene delivery delays onset and severity of collagen II arthritis.

Gjertsson I, Laurie KL, Devitt J, Howe SJ, Thrasher AJ, Holmdahl R, Gustafsson K.

Mol Ther. 2009 Apr;17(4):632-40. doi: 10.1038/mt.2009.299. Epub 2009 Jan 27.

38.

Efficient gene delivery to the adult and fetal CNS using pseudotyped non-integrating lentiviral vectors.

Rahim AA, Wong AM, Howe SJ, Buckley SM, Acosta-Saltos AD, Elston KE, Ward NJ, Philpott NJ, Cooper JD, Anderson PN, Waddington SN, Thrasher AJ, Raivich G.

Gene Ther. 2009 Apr;16(4):509-20. doi: 10.1038/gt.2008.186. Epub 2009 Jan 22.

PMID:
19158847
39.

Haematopoietic repopulating activity in human cord blood CD133+ quiescent cells.

Boxall SA, Cook GP, Pearce D, Bonnet D, El-Sherbiny YM, Blundell MP, Howe SJ, Leek JP, Markham AF, de Wynter EA.

Bone Marrow Transplant. 2009 Apr;43(8):627-35. doi: 10.1038/bmt.2008.368. Epub 2008 Nov 10.

PMID:
18997827
40.

Luciferin detection after intranasal vector delivery is improved by intranasal rather than intraperitoneal luciferin administration.

Buckley SM, Howe SJ, Rahim AA, Buning H, McIntosh J, Wong SP, Baker AH, Nathwani A, Thrasher AJ, Coutelle C, McKay TR, Waddington SN.

Hum Gene Ther. 2008 Oct;19(10):1050-6. doi: 10.1089/hum.2008.023.

PMID:
18847316
41.

Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients.

Howe SJ, Mansour MR, Schwarzwaelder K, Bartholomae C, Hubank M, Kempski H, Brugman MH, Pike-Overzet K, Chatters SJ, de Ridder D, Gilmour KC, Adams S, Thornhill SI, Parsley KL, Staal FJ, Gale RE, Linch DC, Bayford J, Brown L, Quaye M, Kinnon C, Ancliff P, Webb DK, Schmidt M, von Kalle C, Gaspar HB, Thrasher AJ.

J Clin Invest. 2008 Sep;118(9):3143-50. doi: 10.1172/JCI35798.

42.

Persistent episomal transgene expression in liver following delivery of a scaffold/matrix attachment region containing non-viral vector.

Argyros O, Wong SP, Niceta M, Waddington SN, Howe SJ, Coutelle C, Miller AD, Harbottle RP.

Gene Ther. 2008 Dec;15(24):1593-605. doi: 10.1038/gt.2008.113. Epub 2008 Jul 17.

PMID:
18633447
43.

Lentiviral transduction of the murine lung provides efficient pseudotype and developmental stage-dependent cell-specific transgene expression.

Buckley SM, Howe SJ, Sheard V, Ward NJ, Coutelle C, Thrasher AJ, Waddington SN, McKay TR.

Gene Ther. 2008 Aug;15(16):1167-75. doi: 10.1038/gt.2008.74. Epub 2008 Apr 24.

PMID:
18432275
44.

Self-inactivating gammaretroviral vectors for gene therapy of X-linked severe combined immunodeficiency.

Thornhill SI, Schambach A, Howe SJ, Ulaganathan M, Grassman E, Williams D, Schiedlmeier B, Sebire NJ, Gaspar HB, Kinnon C, Baum C, Thrasher AJ.

Mol Ther. 2008 Mar;16(3):590-8. doi: 10.1038/sj.mt.6300393. Epub 2008 Jan 8.

45.

Cell-specific and efficient expression in mouse and human B cells by a novel hybrid immunoglobulin promoter in a lentiviral vector.

Laurie KL, Blundell MP, Baxendale HE, Howe SJ, Sinclair J, Qasim W, Brunsberg U, Thrasher AJ, Holmdahl R, Gustafsson K.

Gene Ther. 2007 Dec;14(23):1623-31. Epub 2007 Sep 13.

PMID:
17851547
46.

Gammaretrovirus-mediated correction of SCID-X1 is associated with skewed vector integration site distribution in vivo.

Schwarzwaelder K, Howe SJ, Schmidt M, Brugman MH, Deichmann A, Glimm H, Schmidt S, Prinz C, Wissler M, King DJ, Zhang F, Parsley KL, Gilmour KC, Sinclair J, Bayford J, Peraj R, Pike-Overzet K, Staal FJ, de Ridder D, Kinnon C, Abel U, Wagemaker G, Gaspar HB, Thrasher AJ, von Kalle C.

J Clin Invest. 2007 Aug;117(8):2241-9.

47.

Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy.

Deichmann A, Hacein-Bey-Abina S, Schmidt M, Garrigue A, Brugman MH, Hu J, Glimm H, Gyapay G, Prum B, Fraser CC, Fischer N, Schwarzwaelder K, Siegler ML, de Ridder D, Pike-Overzet K, Howe SJ, Thrasher AJ, Wagemaker G, Abel U, Staal FJ, Delabesse E, Villeval JL, Aronow B, Hue C, Prinz C, Wissler M, Klanke C, Weissenbach J, Alexander I, Fischer A, von Kalle C, Cavazzana-Calvo M.

J Clin Invest. 2007 Aug;117(8):2225-32.

48.

Lentiviral vectors containing an enhancer-less ubiquitously acting chromatin opening element (UCOE) provide highly reproducible and stable transgene expression in hematopoietic cells.

Zhang F, Thornhill SI, Howe SJ, Ulaganathan M, Schambach A, Sinclair J, Kinnon C, Gaspar HB, Antoniou M, Thrasher AJ.

Blood. 2007 Sep 1;110(5):1448-57. Epub 2007 Apr 24.

49.

Ectopic retroviral expression of LMO2, but not IL2Rgamma, blocks human T-cell development from CD34+ cells: implications for leukemogenesis in gene therapy.

Pike-Overzet K, de Ridder D, Weerkamp F, Baert MR, Verstegen MM, Brugman MH, Howe SJ, Reinders MJ, Thrasher AJ, Wagemaker G, van Dongen JJ, Staal FJ.

Leukemia. 2007 Apr;21(4):754-63. Epub 2007 Feb 1.

PMID:
17268520
50.

Gene therapy: is IL2RG oncogenic in T-cell development?

Pike-Overzet K, de Ridder D, Weerkamp F, Baert MR, Verstegen MM, Brugman MH, Howe SJ, Reinders MJ, Thrasher AJ, Wagemaker G, van Dongen JJ, Staal FJ.

Nature. 2006 Sep 21;443(7109):E5; discussion E6-7.

PMID:
16988660

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