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Items: 45


Gene Therapy for Parkinson's Disease: Preclinical Evaluation of Optimally Configured TH:CH1 Fusion for Maximal Dopamine Synthesis.

Badin RA, Binley K, Van Camp N, Jan C, Gourlay J, Robert C, Gipchtein P, Fayard A, Stewart H, Ralph GS, Lad Y, Kelleher M, Loader J, Hosomi K, Palfi S, Mitrophanous KA, Hantraye P.

Mol Ther Methods Clin Dev. 2019 Jul 16;14:206-216. doi: 10.1016/j.omtm.2019.07.002. eCollection 2019 Sep 13.


Long-Term Follow-Up of a Phase I/II Study of ProSavin, a Lentiviral Vector Gene Therapy for Parkinson's Disease.

Palfi S, Gurruchaga JM, Lepetit H, Howard K, Ralph GS, Mason S, Gouello G, Domenech P, Buttery PC, Hantraye P, Tuckwell NJ, Barker RA, Mitrophanous KA.

Hum Gene Ther Clin Dev. 2018 Sep;29(3):148-155. doi: 10.1089/humc.2018.081.


Optogenetic Tractography for anatomo-functional characterization of cortico-subcortical neural circuits in non-human primates.

Senova S, Poupon C, Dauguet J, Stewart HJ, Dugué GP, Jan C, Hosomi K, Ralph GS, Barnes L, Drouot X, Pouzat C, Mangin JF, Pain F, Doignon I, Aron-Badin R, Brouillet E, Boyden ES, Mitrophanous KA, Hantraye P, Palfi S.

Sci Rep. 2018 Feb 20;8(1):3362. doi: 10.1038/s41598-018-21486-8.


Safety and Efficacy of OXB-202, a Genetically Engineered Tissue Therapy for the Prevention of Rejection in High-Risk Corneal Transplant Patients.

Fouladi N, Parker M, Kennedy V, Binley K, McCloskey L, Loader J, Kelleher M, Mitrophanous KA, Stout JT, Ellis S.

Hum Gene Ther. 2018 Jun;29(6):687-698. doi: 10.1089/hum.2017.184. Epub 2018 Mar 19.


Enhancing titres of therapeutic viral vectors using the transgene repression in vector production (TRiP) system.

Maunder HE, Wright J, Kolli BR, Vieira CR, Mkandawire TT, Tatoris S, Kennedy V, Iqball S, Devarajan G, Ellis S, Lad Y, Clarkson NG, Mitrophanous KA, Farley DC.

Nat Commun. 2017 Mar 27;8:14834. doi: 10.1038/ncomms14834.


Separate elements of episodic memory subserved by distinct hippocampal-prefrontal connections.

Barker GR, Banks PJ, Scott H, Ralph GS, Mitrophanous KA, Wong LF, Bashir ZI, Uney JB, Warburton EC.

Nat Neurosci. 2017 Feb;20(2):242-250. doi: 10.1038/nn.4472. Epub 2017 Jan 9.


Lentiviral Vector Gene Transfer of Endostatin/Angiostatin for Macular Degeneration (GEM) Study.

Campochiaro PA, Lauer AK, Sohn EH, Mir TA, Naylor S, Anderton MC, Kelleher M, Harrop R, Ellis S, Mitrophanous KA.

Hum Gene Ther. 2017 Jan;28(1):99-111. doi: 10.1089/hum.2016.117. Epub 2016 Sep 26.


Optimizing Transgene Configuration and Protein Fusions to Maximize Dopamine Production for the Gene Therapy of Parkinson's Disease.

Stewart HJ, Ralph GS, Fong-Wong L, Strickland I, McCloskey L, Barnes L, Blount I, Wells O, Truran CJ, Kingsman AJ, Palfi S, Mitrophanous KA.

Hum Gene Ther Clin Dev. 2016 Sep;27(3):100-10. doi: 10.1089/humc.2016.056. Epub 2016 Jul 28.


Development of a replication-competent lentivirus assay for dendritic cell-targeting lentiviral vectors.

Farley DC, McCloskey L, Thorne BA, Tareen SU, Nicolai CJ, Campbell DJ, Bannister R, Stewart HJ, Pearson LJ, Moyer BJ, Robbins SH, Zielinski L, Kim T, Radcliffe PA, Mitrophanous KA, Gombotz WR, Miskin JE, Kelley-Clarke B.

Mol Ther Methods Clin Dev. 2015 May 13;2:15017. doi: 10.1038/mtm.2015.17. eCollection 2015.


EIAV-based retinal gene therapy in the shaker1 mouse model for usher syndrome type 1B: development of UshStat.

Zallocchi M, Binley K, Lad Y, Ellis S, Widdowson P, Iqball S, Scripps V, Kelleher M, Loader J, Miskin J, Peng YW, Wang WM, Cheung L, Delimont D, Mitrophanous KA, Cosgrove D.

PLoS One. 2014 Apr 4;9(4):e94272. doi: 10.1371/journal.pone.0094272. eCollection 2014.


Suppression of neovascularization of donor corneas by transduction with equine infectious anemia virus-based lentiviral vectors expressing endostatin and angiostatin.

Parker M, Bellec J, McFarland T, Scripps V, Appukuttan B, Hartzell M, Yeager A, Hady T, Mitrophanous KA, Stout T, Ellis S.

Hum Gene Ther. 2014 May;25(5):408-18. doi: 10.1089/hum.2013.079. Epub 2014 Mar 21.


Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1/2 trial.

Palfi S, Gurruchaga JM, Ralph GS, Lepetit H, Lavisse S, Buttery PC, Watts C, Miskin J, Kelleher M, Deeley S, Iwamuro H, Lefaucheur JP, Thiriez C, Fenelon G, Lucas C, Brugières P, Gabriel I, Abhay K, Drouot X, Tani N, Kas A, Ghaleh B, Le Corvoisier P, Dolphin P, Breen DP, Mason S, Guzman NV, Mazarakis ND, Radcliffe PA, Harrop R, Kingsman SM, Rascol O, Naylor S, Barker RA, Hantraye P, Remy P, Cesaro P, Mitrophanous KA.

Lancet. 2014 Mar 29;383(9923):1138-46. doi: 10.1016/S0140-6736(13)61939-X. Epub 2014 Jan 10.


Transduction of photoreceptors with equine infectious anemia virus lentiviral vectors: safety and biodistribution of StarGen for Stargardt disease.

Binley K, Widdowson P, Loader J, Kelleher M, Iqball S, Ferrige G, de Belin J, Carlucci M, Angell-Manning D, Hurst F, Ellis S, Miskin J, Fernandes A, Wong P, Allikmets R, Bergstrom C, Aaberg T, Yan J, Kong J, Gouras P, Prefontaine A, Vezina M, Bussieres M, Naylor S, Mitrophanous KA.

Invest Ophthalmol Vis Sci. 2013 Jun 12;54(6):4061-71. doi: 10.1167/iovs.13-11871.


Characterization of lentiviral vector production using microwell suspension cultures of HEK293T-derived producer cells.

Guy HM, McCloskey L, Lye GJ, Mitrophanous KA, Mukhopadhyay TK.

Hum Gene Ther Methods. 2013 Apr;24(2):125-39. doi: 10.1089/hgtb.2012.200.


Assessment of Integration-defective HIV-1 and EIAV Vectors In Vitro and In Vivo.

Ellis S, Fong-Wong L, Iqball S, Thoree V, Mitrophanous KA, Binley K.

Mol Ther Nucleic Acids. 2012 Dec 11;1:e60. doi: 10.1038/mtna.2012.53.


Development of an equine-tropic replication-competent lentivirus assay for equine infectious anemia virus-based lentiviral vectors.

Farley DC, Bannister R, Leroux-Carlucci MA, Evans NE, Miskin JE, Mitrophanous KA.

Hum Gene Ther Methods. 2012 Oct;23(5):309-23. doi: 10.1089/hgtb.2012.102. Epub 2012 Nov 2.


A stable producer cell line for the manufacture of a lentiviral vector for gene therapy of Parkinson's disease.

Stewart HJ, Fong-Wong L, Strickland I, Chipchase D, Kelleher M, Stevenson L, Thoree V, McCarthy J, Ralph GS, Mitrophanous KA, Radcliffe PA.

Hum Gene Ther. 2011 Mar;22(3):357-69. doi: 10.1089/hum.2010.142. Epub 2011 Feb 10.


Dopamine gene therapy for Parkinson's disease in a nonhuman primate without associated dyskinesia.

Jarraya B, Boulet S, Ralph GS, Jan C, Bonvento G, Azzouz M, Miskin JE, Shin M, Delzescaux T, Drouot X, Hérard AS, Day DM, Brouillet E, Kingsman SM, Hantraye P, Mitrophanous KA, Mazarakis ND, Palfi S.

Sci Transl Med. 2009 Oct 14;1(2):2ra4. doi: 10.1126/scitranslmed.3000130.


Development of inducible EIAV-based lentiviral vector packaging and producer cell lines.

Stewart HJ, Leroux-Carlucci MA, Sion CJ, Mitrophanous KA, Radcliffe PA.

Gene Ther. 2009 Jun;16(6):805-14. doi: 10.1038/gt.2009.20. Epub 2009 Mar 5.


Localised axial progenitor cell populations in the avian tail bud are not committed to a posterior Hox identity.

McGrew MJ, Sherman A, Lillico SG, Ellard FM, Radcliffe PA, Gilhooley HJ, Mitrophanous KA, Cambray N, Wilson V, Sang H.

Development. 2008 Jul;135(13):2289-99. doi: 10.1242/dev.022020. Epub 2008 May 28.


Analysis of factor VIII mediated suppression of lentiviral vector titres.

Radcliffe PA, Sion CJ, Wilkes FJ, Custard EJ, Beard GL, Kingsman SM, Mitrophanous KA.

Gene Ther. 2008 Feb;15(4):289-97. Epub 2007 Nov 29.


Factors that influence VSV-G pseudotyping and transduction efficiency of lentiviral vectors-in vitro and in vivo implications.

Farley DC, Iqball S, Smith JC, Miskin JE, Kingsman SM, Mitrophanous KA.

J Gene Med. 2007 May;9(5):345-56.


Oviduct-specific expression of two therapeutic proteins in transgenic hens.

Lillico SG, Sherman A, McGrew MJ, Robertson CD, Smith J, Haslam C, Barnard P, Radcliffe PA, Mitrophanous KA, Elliot EA, Sang HM.

Proc Natl Acad Sci U S A. 2007 Feb 6;104(6):1771-6. Epub 2007 Jan 26.


The integration profile of EIAV-based vectors.

Hacker CV, Vink CA, Wardell TW, Lee S, Treasure P, Kingsman SM, Mitrophanous KA, Miskin JE.

Mol Ther. 2006 Oct;14(4):536-45.


Lentivirus-mediated gene transfer to the central nervous system: therapeutic and research applications.

Wong LF, Goodhead L, Prat C, Mitrophanous KA, Kingsman SM, Mazarakis ND.

Hum Gene Ther. 2006 Jan;17(1):1-9. Review. Erratum in: Hum Gene Ther. 2006 Mar;17(3):376.


In vivo evaluation of an EIAV vector for the systemic genetic delivery of therapeutic antibodies.

Lamikanra A, Myers KA, Ferris N, Mitrophanous KA, Carroll MW.

Gene Ther. 2005 Jun;12(12):988-98.


Silencing mutant SOD1 using RNAi protects against neurodegeneration and extends survival in an ALS model.

Ralph GS, Radcliffe PA, Day DM, Carthy JM, Leroux MA, Lee DC, Wong LF, Bilsland LG, Greensmith L, Kingsman SM, Mitrophanous KA, Mazarakis ND, Azzouz M.

Nat Med. 2005 Apr;11(4):429-33. Epub 2005 Mar 13.


Lentivector-mediated SMN replacement in a mouse model of spinal muscular atrophy.

Azzouz M, Le T, Ralph GS, Walmsley L, Monani UR, Lee DC, Wilkes F, Mitrophanous KA, Kingsman SM, Burghes AH, Mazarakis ND.

J Clin Invest. 2004 Dec;114(12):1726-31.


Efficient generation of transgenic pigs using equine infectious anaemia virus (EIAV) derived vector.

Whitelaw CB, Radcliffe PA, Ritchie WA, Carlisle A, Ellard FM, Pena RN, Rowe J, Clark AJ, King TJ, Mitrophanous KA.

FEBS Lett. 2004 Jul 30;571(1-3):233-6.


Efficient production of germline transgenic chickens using lentiviral vectors.

McGrew MJ, Sherman A, Ellard FM, Lillico SG, Gilhooley HJ, Kingsman AJ, Mitrophanous KA, Sang H.

EMBO Rep. 2004 Jul;5(7):728-33. Epub 2004 Jun 11.


VEGF delivery with retrogradely transported lentivector prolongs survival in a mouse ALS model.

Azzouz M, Ralph GS, Storkebaum E, Walmsley LE, Mitrophanous KA, Kingsman SM, Carmeliet P, Mazarakis ND.

Nature. 2004 May 27;429(6990):413-7.


Highly efficient EIAV-mediated in utero gene transfer and expression in the major muscle groups affected by Duchenne muscular dystrophy.

Gregory LG, Waddington SN, Holder MV, Mitrophanous KA, Buckley SM, Mosley KL, Bigger BW, Ellard FM, Walmsley LE, Lawrence L, Al-Allaf F, Kingsman S, Coutelle C, Themis M.

Gene Ther. 2004 Jul;11(14):1117-25.


Neuroprotection in a rat Parkinson model by GDNF gene therapy using EIAV vector.

Azzouz M, Ralph S, Wong LF, Day D, Askham Z, Barber RD, Mitrophanous KA, Kingsman SM, Mazarakis ND.

Neuroreport. 2004 Apr 29;15(6):985-90.


Transduction patterns of pseudotyped lentiviral vectors in the nervous system.

Wong LF, Azzouz M, Walmsley LE, Askham Z, Wilkes FJ, Mitrophanous KA, Kingsman SM, Mazarakis ND.

Mol Ther. 2004 Jan;9(1):101-11. Erratum in: Mol Ther. 2004 May;9(5):765.


Long-term transgene expression by administration of a lentivirus-based vector to the fetal circulation of immuno-competent mice.

Waddington SN, Mitrophanous KA, Ellard FM, Buckley SM, Nivsarkar M, Lawrence L, Cook HT, Al-Allaf F, Bigger B, Kingsman SM, Coutelle C, Themis M.

Gene Ther. 2003 Aug;10(15):1234-40.


Long-term replacement of a mutated nonfunctional CNS gene: reversal of hypothalamic diabetes insipidus using an EIAV-based lentiviral vector expressing arginine vasopressin.

Bienemann AS, Martin-Rendon E, Cosgrave AS, Glover CP, Wong LF, Kingsman SM, Mitrophanous KA, Mazarakis ND, Uney JB.

Mol Ther. 2003 May;7(5 Pt 1):588-96.


Retinoic acid receptor beta2 and neurite outgrowth in the adult mouse spinal cord in vitro.

Corcoran J, So PL, Barber RD, Vincent KJ, Mazarakis ND, Mitrophanous KA, Kingsman SM, Maden M.

J Cell Sci. 2002 Oct 1;115(Pt 19):3779-86.


Gene transduction efficiency in cells of different species by HIV and EIAV vectors.

Ikeda Y, Collins MK, Radcliffe PA, Mitrophanous KA, Takeuchi Y.

Gene Ther. 2002 Jul;9(14):932-8.


New methods to titrate EIAV-based lentiviral vectors.

Martin-Rendon E, White LJ, Olsen A, Mitrophanous KA, Mazarakis ND.

Mol Ther. 2002 May;5(5 Pt 1):566-70.


Design, production, safety, evaluation, and clinical applications of nonprimate lentiviral vectors.

Rohll JB, Mitrophanous KA, Martin-Rendon E, Ellard FM, Radcliffe PA, Mazarakis ND, Kingsman SM.

Methods Enzymol. 2002;346:466-500. No abstract available.


Rabies virus glycoprotein pseudotyping of lentiviral vectors enables retrograde axonal transport and access to the nervous system after peripheral delivery.

Mazarakis ND, Azzouz M, Rohll JB, Ellard FM, Wilkes FJ, Olsen AL, Carter EE, Barber RD, Baban DF, Kingsman SM, Kingsman AJ, O'Malley K, Mitrophanous KA.

Hum Mol Genet. 2001 Sep 15;10(19):2109-21.


Characterization of the equine infectious anaemia virus S2 protein.

Yoon S, Kingsman SM, Kingsman AJ, Wilson SA, Mitrophanous KA.

J Gen Virol. 2000 Sep;81(Pt 9):2189-94.


Growth factor enhanced retroviral gene transfer to the adult central nervous system.

King LA, Mitrophanous KA, Clark LA, Kim VN, Rohll JB, Kingsman AJ, Colello RJ.

Gene Ther. 2000 Jul;7(13):1103-11.


A Rev-independent human immunodeficiency virus type 1 (HIV-1)-based vector that exploits a codon-optimized HIV-1 gag-pol gene.

Kotsopoulou E, Kim VN, Kingsman AJ, Kingsman SM, Mitrophanous KA.

J Virol. 2000 May;74(10):4839-52.

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