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Targeted gene therapy in radiotherapy

: activation by ionizing radiation or uptake of radioactive iododeoxyuridine. Hum Gene Ther 1998; 9(10) : 1409-17. Weichselbaum RR, Hallahan DE, Beckett MA, Mauceri HJ, Lee H, Sukhatme VP, Kufe DW. Gene therapy targeted by radiation preferentially radiosensitizes tumor cells. Cancer Res 1994; 54(16) : 4266-9. Hallahan DE, Mauceri HJ, Seung LP, Dunphy EJ, Wayne JD, Hanna NN, Toledano A, Hellman S, Kufe DW, Weichselbaum RR. Spatial and temporal control of gene therapy using ionizing radiation. Nat Med 1995; 1

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Gene therapy for haemophilia: a very modern success story

References 1. Morris J. Patient advocacy helps patients weigh up gene therapy trial risk/benefits. J Haem Pract 2015; 2(1): 6-8. doi: 10.17225/jhp.00040. Avaialble at http://www.haemjournal.com/external/articles/doi/10.17225/jhp.00040 2. Nathwani AC, Tuddenham EG, Rangarajan S, et al. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N Engl J Med 2011; 365(25): 2357-65. doi: 10.1056/NEJMoa1108046.

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Gene therapy for haemophilia: an update on progress in clinical trials

References 1. Cancio MI, Reiss UM, Nathwani AC, et al. Developments in the treatment of hemophilia B: focus on emerging gene therapy. Appl Clin Genet 2013; 6: 91-101. doi: 10.2147/TACG.S31928. 2. Gringeri A, Ewenstein B, Reininger A. The burden of bleeding in haemophilia: is one bleed too many? Haemophilia 2014; 20(4): 459-63. doi: 10.1111/hae.12375. 3. Schrijvers LH, Uitslager N, Schuurmans MJ, Fischer K.Barriers and motivators of adherence to prophylactic treatment in haemophilia: a systematic review

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Do fair and just systems require compensation for the disadvantages of the natural lottery? a discussion on society's duties on the provision of gene therapy

. Research on genetic disorders such as LPLD and ADA-SCID aim to develop treatments for these kinds of genetic diseases that have high rates of morbidity and mortality. Alipogene tiparvovec (brand name Glybera), is a gene therapy product indicated for the treatment of LPLD, which was approved for use in the European Union on March 8, 2004 [ 1 ], and to date has only successfully treated one patient. After Glybera, Strimvelis was the second gene therapy approved by European Medicines Agency (EMEA) for the treatment of ADA-SCID [ 2 ]. It was the first instance of marketing

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Patient advocacy helps patients weigh up gene therapy trial risk/benefits

References 1. Nathwani AC, Tuddenham EG, Rangarajan S, et al. AdenovirusPassociated virus vectorPmediated gene transfer in hemophilia B. N Engl J Med 2011; 365: 2357P65. doi: 10.1056/NEJMoa1108046. 2. Nathwani AC, Reiss UM, Tuddenham EG, et al. LongPterm safety and efficacy of factor IX gene therapy in hemophilia B. N Engl J Med 2014; 371: 1994P2004. doi: 10.1056/NEJMoa1407309.

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The impending medical revolution in haemophilia care: one patient’s view

patients with Hemophilia B. TOnlineU Available from: URL http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm391037.htmRLast accessed 12/12/2014S. 9. Roosendaal G, Lafeber FP. Pathogenesis of haemophilic arthropathy. Haemophilia 2006; 12: RSuppl. 3S, 117V121. 10. Cancio MI, Reiss UM, Nathwani AC, et al. Developments in the treatment of hemophilia B: focus on emerging gene therapy. Appl Clin Genet 2013; 6: 91Q101. doi: 10.2147/TACG.S31928. 11. High KH, Nathwani A, Spencer T, Lillicrap D. Current status of

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A glance on recent progresses in diagnosis and treatment of primary immunodeficiencies/ Progrese recente în diagnosticul şi tratamentul imunodeficienţelor primare

cells: functional correction by zinc finger nuclease-mediated safe harbor targeting. Blood. 2011 May 26;117(21):5561-72. DOI: 10.1182/blood-2010-12-328161 89. Cavazzana-Calvo M, Fischer A, Hacein-Bey-Abina S, Aiuti A. Gene therapy for primary immunodeficiencies: Part 1. Curr Opin Immunol 2012 Oct;24(5):580-4. DOI: 10.1016/j.coi.2012.08.008 90. Aiuti A, Bacchetta R, Seger R, Villa A, Cavazzana-Calvo M. Gene therapy for primary immunodeficiencies: Part 2. Curr Opin Immunol 2012 Oct;24(5):585-91. DOI: 10.1016/j.coi.2012

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Molecular Genetic Markers as a Basis for Personalized Medicine / MOLEKULARNO-GENETIČKI MARKERI KAO OSNOV ZA PERSONALIZOVANU MEDICINU

oblimersend sodium an antisense to Bcl2 in untreated older patients with AML. J Clin Oncol 2005; 23: 3404-11. 107. MacConaill LE, Garraway LA. Clinical Implications of the Cancer Genome. J Clin Oncol 2010; 28: 5219-28. 108. Anderson WF. Prospects for human gene therapy. Science 1984; 226: 401-9. 109. Friedmann T. Progress toward human gene therapy. Science 1989; 244: 1275-81. 110. Yamamoto M, Curiel DT. Cancer gene therapy. Technol Cancer Res Treat 2005; 4(4): 315-30. 111. Goff S, Berg P

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Electrotransfer of plasmid DNA radiosensitizes B16F10 tumors through activation of immune response

-62. 10.1016/bs.adgen.2014.10.006 Heller R Heller LC. Gene electrotransfer clinical trials Adv Genet 2015 89 235 62 10.1016/bs.adgen.2014.10.006 6 Heller LC, Heller R. Electroporation gene therapy preclinical and clinical trials for melanoma. Curr Gene Ther 2010; 10 : 312-7. 10.2174/156652310791823489 10.2174/156652310791823489 Heller LC Heller R. Electroporation gene therapy preclinical and clinical trials for melanoma Curr Gene Ther 2010 10 312 7 10.2174/156652310791823489 7 Daud AI, DeConti RC, Andrews S, Urbas P

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