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Human Gene Therapy, 1043-0342

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

    Di, W. L., Lwin, S. M., Petrova, A., Bernadis, C., Syed, F., Farzaneh, F., Moulding, D., Martinez, A. E., Sebire, N. J., Rampling, D., Virasami, A., Zamiri, M., Wang, W., Hara, H., Kadiyirire, T., Abdul-Wahab, A., Martinez-Queipo, M., Harper, J. I., McGrath, J. A., Thrasher, A. J. & 2 others, Mellerio, J. E. & Qasim, W., 17 Sep 2019, In : Human Gene Therapy. 30, 9, p. 1067-1078 12 p.

    Research output: Contribution to journalArticle

  2. An Improved Adeno-Associated Virus Vector for Neurological Correction of the Mouse Model of Mucopolysaccharidosis IIIA

    Gray, A. L., O'Leary, C., Liao, A., Agúndez, L., Youshani, A. S., Gleitz, H. F., Parker, H., Taylor, J. T., Danos, O., Hocquemiller, M., Palomar, N., Linden, R. M., Henckaerts, E., Holley, R. J. & Bigger, B. W., 1 Sep 2019, In : Human Gene Therapy. 30, 9, p. 1052-1066 15 p.

    Research output: Contribution to journalArticle

  3. Preferential targeting of disseminated liver tumours using a recombinant adeno-associated virus vector

    Della, P. M., Badar, A., Rosales, C., Chokshi, A., Nathwani, G. E., Yan, R., Arstad, E., Davidoff, AM., Williams, R., Lythgoe, MF. & Nathwani, AC., 2015, In : Human Gene Therapy. p. 94-103 Feb;26(2).

    Research output: Contribution to journalArticle

  4. Partial Correction of the CNS Lysosomal Storage Defect in a Mouse Model of Juvenile Neuronal Ceroid Lipofuscinosis by Neonatal CNS Administration of an Adeno-Associated Virus Serotype rh. 10 Vector Expressing the Human CLN3 Gene

    Sondhi, D., Scott, E. C., Chen, A., Hackett, N. R., Wong, A. M. S., Kubiak, A., Nelvagal, H. R., Pearse, Y., Cotman, S. L., Cooper, J. D. & Crystal, R. G., 1 Mar 2014, In : Human Gene Therapy. 25, 3, p. 223-239 17 p.

    Research output: Contribution to journalArticle

  5. Life-long correction of hyperbilirubinemia with a neonatal liver-specific AAV-mediated gene transfer in a lethal mouse model of Crigler-Najjar syndrome

    Bortolussi, G., Zentillin, L., Vaníkova, J., Bockor, L., Bellarosa, C., Mancarella, A., Vianello, E., Tiribelli, C., Giacca, M., Vitek, L. & Muro, A. F., 1 Jan 2014, In : Human Gene Therapy. 25, 9, p. 844-855 12 p.

    Research output: Contribution to journalArticle

  6. Manufacturing and characterization of a recombinant adeno-associated virus type 8 reference standard material

    Ayuso, E., Blouin, V., Lock, M., Mcgorray, S., Leon, X., Alvira, M. R., Auricchio, A., Bucher, S., Chtarto, A., Clark, K. R., Darmon, C., Doria, M., Fountain, W., Gao, G., Gao, K., Giacca, M., Kleinschmidt, J., Leuchs, B., Melas, C., Mizukami, H. & 16 others, Müller, M., Noordman, Y., Bockstael, O., Ozawa, K., Pythoud, C., Sumaroka, M., Surosky, R., Tenenbaum, L., Van Der Linden, I., Weins, B., Wright, J. F., Zhang, X., Zentilin, L., Bosch, F., Snyder, R. O. & Moullier, P., 1 Jan 2014, In : Human Gene Therapy. 25, 11, p. 977-987 11 p.

    Research output: Contribution to journalArticle

  7. Phase I study protocol for ex vivo lentiviral gene therapy for the inherited skin disease, netherton syndrome

    Di, W-L., Mellerio, J. E., Bernadis, C., Harper, J., Abdul-Wahab, A., Ghani, S., Chan, L., Martinez-Queipo, M., Hara, H., McNicol, A-M., Farzaneh, F., McGrath, J., Thrasher, A. & Qasim, W., 11 Dec 2013, In : Human Gene Therapy. 24, 4, p. 182-190 9 p., N/A.

    Research output: Contribution to journalArticle

  8. Efficient striatal expression of hGDNF from integration-deficient lentiviral vectors ameliorates 6-OHDA-lesioned rats

    Lu-Nguyen, N., Broadstock, M. & Yanez-Munoz, R. J., May 2013, In : Human Gene Therapy. 24, 5, p. A4-A4 1 p., FB4.

    Research output: Contribution to journalMeeting abstract

  9. Optimizing retroviral gene expression for effective therapies

    Antoniou, M., Skipper, K. A. & Anakok, O., Apr 2013, In : Human Gene Therapy. 24, 4, p. 363-374 11 p.

    Research output: Contribution to journalArticle

  10. Development of a gene expression system for enhanced erythroid expression

    Montiel-Equihua, C. A., Zhang, L., Knight, S., Saadeh, H., Scholz, S., Carmo, M., Alonso-Ferrero, M. E., Blundell, M. P., Monkeviciute, A., Schulz, R., Collins, M., Takeuchi, Y., Schmidt, M., Fairbanks, L., Antoniou, M., Thrasher, A. J. & Gaspar, H. B., May 2012, In : Human Gene Therapy. 23, 5, p. A11-A11 1 p., P 012.

    Research output: Contribution to journalPoster abstract

  11. Challenges for Gene Therapy of CNS Disorders and Implications for Parkinson's Disease Therapies

    Broadstock, M. & Yanez-Munoz, R. J., 19 Apr 2012, In : Human Gene Therapy. 23, 4, p. 340-343 4 p., N/A.

    Research output: Contribution to journalEditorial

  12. Enhanced athletic performance on multisite AAV-IGF1 gene transfer coincides with massive modification of the muscle proteome

    MacEdo, A., Moriggi, M., Vasso, M., De Palma, S., Sturnega, M., Friso, G., Gelfi, C., Giacca, M. & Zacchigna, S., 1 Feb 2012, In : Human Gene Therapy. 23, 2, p. 146-157 12 p.

    Research output: Contribution to journalArticle

  13. In Vitro Primary Cell Culture as a Physiologically Relevant Method for Preclinical Testing of Human Oncolytic Adenovirus

    Adamson, R. E., Frazier, A. A., Evans, H., Chambers, K. F., Schenk, E., Essand, M., Birnie, R., Mitry, R. R., Dhawan, A. & Maitland, N. J., 1 Feb 2012, In : Human Gene Therapy. 23, 2, p. 218 - 230 13 p.

    Research output: Contribution to journalArticle

  14. Critical Physiological and Surgical Considerations for Hydrodynamic Pressurization of Individual Segments of the Pig Liver

    Fabre, J. W., Whitehorne, M., Grehan, A., Sawyer, G. J., Zhang, X., Davenport, M. & Rela, M., 14 Jul 2011, In : Human Gene Therapy. 22, 7, p. 879 - 887 9 p.

    Research output: Contribution to journalArticle

  15. Delivering on RNAi Therapeutics, Molecular Imaging and Theranostics; what do we think that we know?

    Thanou, M. & Miller, A. D., 2011, In : Human Gene Therapy. 22, 6, p. A8 - A8

    Research output: Contribution to journalMeeting abstract

  16. Characterization of a recombinant adeno-associated virus type 2 reference standard material

    Lock, M., McGorray, S., Auricchio, A., Ayuso, E., Beecham, E. J., Blouin-Tavel, V., Bosch, F., Bose, M., Byrne, B. J., Caton, T., Chiorini, J. A., Chtarto, A., Clark, K. R., Conlon, T., Darmon, C., Doria, M., Douar, A., Flotte, T. R., Francis, J. D., Francois, A. & 28 others, Giacca, M., Korn, M. T., Korytov, I., Leon, X., Leuchs, B., Lux, G., Melas, C., Mizukami, H., Moullier, P., Müller, M., Ozawa, K., Philipsberg, T., Poulard, K., Raupp, C., Rivière, C., Roosendaal, S. D., Samulski, R. J., Soltys, S. M., Surosky, R., Tenenbaum, L., Thomas, D. L., Van Montfort, B., Veres, G., Wright, J. F., Xu, Y., Zelenaia, O., Zentilin, L. & Snyder, R. O., 1 Oct 2010, In : Human Gene Therapy. 21, 10, p. 1273-1285 13 p.

    Research output: Contribution to journalArticle

  17. International Society for Cell and Gene Therapy of Cancer 2009 Annual Meeting Held in Cork, Ireland

    Guinn, B., Casey, G., Moeller, M. G., Kasahara, N., O'Sullivan, G. C., Peng, K-W. & Tangney, M., 1 Jan 2010, In : Human Gene Therapy. 21, 1, p. 9 - 26 18 p.

    Research output: Contribution to journalLiterature review

  18. A Consortium for Clinical Development of Lentiviral Vector-Induced DCs for Melanoma Immunotherapy

    Stripecke, R., Kuhs, S., Gutzmer, R., Garritsen, H. & Farzaneh, F., 2010, In : Human Gene Therapy. 21, 9, p. 1177 - 1177 1 p.

    Research output: Contribution to journalMeeting abstract

  19. A2UCOE Lentiviral Vector Resists DNA Methylation-Mediated Silencing, Conferring Long-Term Expression in HSCs Following Serial Transplantation In Vivo

    Zhang, F., Antoniou, M. N. & Thrasher, A., 2010, In : Human Gene Therapy. 21, 4, p. 514 - 515 2 p.

    Research output: Contribution to journalMeeting abstract

  20. Conditional Expansion of Human Skeletal Muscle Satellite Cells by the Tamoxifen-Inducible c-mycERTAM Fusion Protein Following Lentiviral Vector Transduction for Cell Therapy Purposes

    Rossi, C. A., Campanella, M., Zia, S., Leclere, P., Rapisarda, C., Pierro, A., Antoniou, M. & De Coppi, P., 2010, In : Human Gene Therapy. 21, 4, p. 523 - 523 1 p.

    Research output: Contribution to journalMeeting abstract

  21. Congenic Fetal Delivered Lentivirus UCOE-Luciferase Transduced Amniotic Fluid Stem Cells Preferentially Engraft in the Liver

    Shaw, S. W. S., David, A. L., Antoniou, M., Meola, M., Ward, N., De Coppi, P. & Waddington, S. N., 2010, In : Human Gene Therapy. 21, 4, p. 522 - 522 1 p.

    Research output: Contribution to journalMeeting abstract

  22. Critical Physiological and Surgical Considerations for Hydrodynamic Gene Delivery to Individual Segments of the Liver in the Rat and Pig Models

    Fabre, J. W., Sawyer, G. J., Whitehorne, M., Grehan, A., Zhang, H., Davenport, M. & Rela, M., 2010, In : Human Gene Therapy. 21, 4, p. 498 - 498 1 p.

    Research output: Contribution to journalMeeting abstract

  23. Development and Assessment of Thermo-Stable Adenovirus (Ad)-Based Vaccines Expressing Ubiquitinated and Genetically Fragmented HIV/SIV Genetic Components

    Athanasopoulos, T., Papagatsias, T., Meiser, A., Harris, J., Benlahrech, A., Herath, S., Roesen, N., Oh, S., Li, F., Self, S., Kwon, S-Y., Fischer, K., Seymour, L., Daniels, R., Almond, N., Klavinskis, L., Logan, K., Patterson, S. & Dickson, G., 2010, In : Human Gene Therapy. 21, 4, p. 520 - 520 1 p.

    Research output: Contribution to journalMeeting abstract

  24. Development of In Utero Lentiviral Vector Gene Therapy Approaches for Inherited Diseases

    Ferreira, S., Kao, V., Waddington, S., Ward, N., Thrasher, A. J. & Antoniou, M. N., 2010, In : Human Gene Therapy. 21, 4, p. 524 - 524 1 p.

    Research output: Contribution to journalMeeting abstract

  25. Efficient CNS, PNS, and Visceral Gene Delivery Following Fetal and Neonatal Intravenous Administration of AAV9

    Rahim, A. A., Wong, A. M., Hoeffer, K., Buckley, S. M., Mattar, C., Chan, J., Cooper, J. D. & Waddington, S. N., 2010, In : Human Gene Therapy. 21, 4, p. 505 - 506 2 p.

    Research output: Contribution to journalMeeting abstract

  26. Efficient Transduction of the Spinal Cord with Integration-Deficient Lentiviral Vectors

    Ahmed, S. G. O., Peluffo, H., Foster, E., Lago, N., Moon, L., Hutson, T., Yip, P., Wanisch, K., Olivan, S., Osta, R., McMahon, S. B. & Yanez-Munoz, R. J., 2010, In : Human Gene Therapy. 21, 4, p. 521 - 521 1 p.

    Research output: Contribution to journalMeeting abstract

  27. Efficient gene expression in the spinal cord from integrase-deficient lentiviral Vectors

    Ahmed, S. O., Peluffo, H., Foster, E., Lago, N., Hutson, T. H., Moon, L., Yip, P., Caraballo-Miralles, V., Llado, J., McMahon, S. B. & Yanez-Munoz, R. J., 2010, In : Human Gene Therapy. 21, 10, p. 1428 - 1428 1 p.

    Research output: Contribution to journalMeeting abstract

  28. Evaluation of Several DNA Expression Constructs for the Level and Longevity of Expression of the Pancreatic Transcription Factor pdx1 in Rat Liver

    Cim, A., Sawyer, G. J., Zhang, H., Su, H., Collins, L., Antoniou, M., Lipps, H. & Fabre, J. W., 2010, In : Human Gene Therapy. 21, 4, p. 517 - 517 1 p.

    Research output: Contribution to journalMeeting abstract

  29. Histidine-rich amphipathic peptides promote efficient delivery of nucleic acids into mammalian cells

    Kichler, A., Mason, A. J., Leborgne, C., Bechinger, B. & Scherman, D., 2010, In : Human Gene Therapy. 21, 10, p. 1413 - 1413 1 p.

    Research output: Contribution to journalMeeting abstract

  30. Immune Gene Therapy for Acute Myeloid Leukaemia

    Farzaneh, F., Chan, L., Hardwick, N., Ingram, W., Mufti, G., Aloysius, M., Robins, A., Habib, N., Bhalla, J., Eremin, O., Jun, T. G., Oppenheim, D., Wells, J., Cowled, C. & Noble, A., 2010, In : Human Gene Therapy. 21, 9, p. 1177 - 1178 2 p.

    Research output: Contribution to journalMeeting abstract

  31. Importance of Neurogenesis in a Rodent Model of Stroke

    Rossetti, T. R., Goncalves, M. B., Yip, P., Doherty, P., Hainsworth, A. H. & Yanez-Munoz, R. J., 2010, In : Human Gene Therapy. 21, 4, p. 520 - 521 2 p.

    Research output: Contribution to journalMeeting abstract

  32. Insertional Mutagenesis by Lentiviral Vectors

    Knight, S. B., Bokhoven, M., Gubta, A., Zhang, F., Antoniou, M., Thrasher, A., Collins, M. & Takeuchi, Y., 2010, In : Human Gene Therapy. 21, 4, p. 496 - 497 2 p.

    Research output: Contribution to journalMeeting abstract

  33. Metabolic Biotinylation of Lentiviral Vectors

    Mondragon, A., Chen, R., Folarin, N., Darling, D., Slater, N. K. H. & Farzaneh, F., 2010, In : Human Gene Therapy. 21, 4, p. 523 - 524 2 p.

    Research output: Contribution to journalMeeting abstract

  34. Monocytes transduced with tricistronic lentiviral vectors in a closed bag system induce differentiation of loaded "SMART-DCs" for melanoma immunotherapy

    Stripecke, R., Kuhs, S., Pincha, M., Gutzmer, R., Salguero, G., Sundarasetty, B., Garritsen, H., Farzaneh, F., Chan, L., Woelfel, T., Schendel, D., Schmiedeknecht, G. & Ganser, A., 2010, In : Human Gene Therapy. 21, 10, p. 1416 - 1416 1 p.

    Research output: Contribution to journalMeeting abstract

  35. Nonviral Delivery of the Rat PDX1 Gene to Rat Liver For the In Vivo Transdifferentiation of Liver Cells to Pancreatic Beta-Cells

    Cim, A., Sawyer, G., Zhang, X., Su, H., Collins, L., Jones, P., Antoniou, M., Lipps, H. J. & Fabre, J., 2010, In : Human Gene Therapy. 21, 9, p. 1182 - 1182 1 p.

    Research output: Contribution to journalMeeting abstract

  36. Perinatal Gene Therapy for Lethal Genetic Diseases

    Waddington, S. N., Rahim, A. A., Mattar, C., Wong, A. M. S., Hoeffer, K., Buckley, S. M. K., Cooper, J. D. & Chan, J., 2010, In : Human Gene Therapy. 21, 9, p. 1174 - 1174 1 p.

    Research output: Contribution to journalMeeting abstract

  37. Systemic Gene Therapy of SCID-ADA: Improving Transgene Expression in the Erythrocyte Lineage

    Montiel-Equihua, C. A., Antoniou, M., Thrasher, A. J. & Gaspar, H. B., 2010, In : Human Gene Therapy. 21, 4, p. 497 - 497 1 p.

    Research output: Contribution to journalMeeting abstract

  38. uPAR Targeted Nanoparticles

    Thanou, M., 2010, In : Human Gene Therapy. 21, 4, p. 488 - 489 2 p.

    Research output: Contribution to journalMeeting abstract

  39. Intestinal Lactase as an Autologous beta-Galactosidase Reporter Gene for In Vivo Gene Expression Studies

    Salehi, S., Eckley, L., Sawyer, G. J., Zhang, X., Dong, X., Freund, J-N. & Fabre, J. W., 1 Jan 2009, In : Human Gene Therapy. 20, 1, p. 21 - 30 10 p.

    Research output: Contribution to journalArticle

  40. Development of SMART-DC co-expressing truncated WT1 for stimulation and expansion of T cells against leukemia relapse

    Sundarasetty, B. S., Rickmann, M., Salguero, G., Kuhs, S., Schambach, A., Farzaneh, F., Herr, W., Ganser, A. & Stripecke, R., 2009, In : Human Gene Therapy. 20, 11, p. 1475 - 1476 2 p.

    Research output: Contribution to journalMeeting abstract

  41. Development of thermo-stable Ad-based vaccines expressing ubiquitin fusions of full or fragmented HIV/SIV genetic components

    Athanasopoulos, T., Papagatsias, T., Meiser, A., Harris, J., Bachy, V., Benlahrech, A., Herath, S., Roesen, N., Oh, S., Li, F., Self, S., Kwon, S-Y., Fischer, K., Seymour, L., Daniels, R., Almond, N., Klavinskis, L., Logan, K., Patterson, S. & Dickson, G., 2009, In : Human Gene Therapy. 20, 11, p. 1392 - 1393 2 p.

    Research output: Contribution to journalMeeting abstract

  42. Targeted gene delivery by AAV

    Linden, M., Dutheil, N. & Henckaerts, E., 2009, In : Human Gene Therapy. 20, 11, p. 1508 - 1508 1 p.

    Research output: Contribution to journalMeeting abstract

  43. A comparative study for efficient transgene expression from non-integrating lentiviral vectors

    Ferreira, S., Leclere, P., Kao, V., Talbot, G. & Antoniou, M., 2008, In : Human Gene Therapy. 19, 4, p. 420 - 420 1 p., 81.

    Research output: Contribution to journalMeeting abstract

  44. Assessment of adenoviral vectors in gel formulations in vitro and in vivo: Aiming towards development of an HIV-1 vaccine

    Harris, J., Athanasopoulos, T., Bachy, V., Papagatsias, T., Benlahrech, A., Csomor, E., Meiser, A., Roesen, N., Oh, S-J., Seymour, L., Fisher, K., Daniels, R., Barbagallo, R., Patterson, S., Klavinskis, L. & Dickson, G., 2008, In : Human Gene Therapy. 19, 10, p. 1142 - 1142 1 p.

    Research output: Contribution to journalMeeting abstract

  45. Comparative analysis of UCOE-based lentiviral vectors in a murine neonatal intravascular delivery system

    Ferreira, S., Waddington, S. & Antoniou, M., 2008, In : Human Gene Therapy. 19, 10, p. 1202 - 1202 1 p.

    Research output: Contribution to journalMeeting abstract

  46. Development of a 'Stealth' adenovirus vaccine vector

    Bachy, V., El-Doueik, H., Fisher, K., Csomor, E., Roesen, N., Seymour, L. & Klavinskis, L., 2008, In : Human Gene Therapy. 19, 4, p. O13

    Research output: Contribution to journalMeeting abstract

  47. Efficient gene delivery to the adult and fetal central nervous system using pseudotyped non-integrating lentiviral vectors

    Rahim, A. A., Waddington, S. N., Wong, A. M. S., Ward, N. J., Elston, K. E., Buckley, S. M. K., Philpott, N. J., Cooper, J. D., Anderson, P. N., Thrasher, A. J. & Raivich, G., 2008, In : Human Gene Therapy. 19, 4, p. O6

    Research output: Contribution to journalMeeting abstract

  48. Evaluation of self-inactivating lentiviral vectors containing myeloid specific promoters in combination with the ubiquitously acting chromatin opening element (UCOE) for gene therapy of X-CGD

    Santilli, G., Blundell, M., Haria, S., Kinnon, C., Antoniou, M., Grez, M. & Thrasher, A., 2008, In : Human Gene Therapy. 19, 4, p. 417 - 417 1 p., 72.

    Research output: Contribution to journalMeeting abstract

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