: 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. Genetherapy 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 genetherapy using ionizing radiation. Nat Med 1995; 1
1. Cancio MI, Reiss UM, Nathwani AC, et al. Developments in the treatment of hemophilia B: focus on emerging genetherapy. 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
. 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 genetherapy 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 genetherapy approved by European Medicines Agency (EMEA) for the treatment of ADA-SCID [ 2 ]. It was the first instance of marketing
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 genetherapy in hemophilia B. N Engl J Med 2014; 371: 1994P2004. doi: 10.1056/NEJMoa1407309.
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 genetherapy. Appl Clin Genet 2013; 6: 91Q101. doi: 10.2147/TACG.S31928.
11. High KH, Nathwani A, Spencer T, Lillicrap D. Current status of
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. Genetherapy 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. Genetherapy for primary immunodeficiencies: Part 2. Curr Opin Immunol 2012 Oct;24(5):585-91. DOI: 10.1016/j.coi.2012
Sonja Pavlović, Branka Zukić and Maja Stojiljković Petrović
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 genetherapy. Science 1984; 226: 401-9.
109. Friedmann T. Progress toward human genetherapy. Science 1989; 244: 1275-81.
110. Yamamoto M, Curiel DT. Cancer genetherapy. Technol Cancer Res Treat 2005; 4(4): 315-30.
111. Goff S, Berg P