Publication: Advanced therapies for hemophilia: reality or fantasy?
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Publication Date
2012-06
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informa healthcare
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Abstract
The article is about the future therapeutic products based on advanced therapies such as gene and cell therapy and tissue engineering or induced pluripotent stem cell technology may offer innumerable potential clinical applicationsfor the treatment of several monogenic
disorders including hemophilia.
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Citation
1 Tirino V, Papaccio G. A new, most likely unusual approach is crucial and upcoming for the use of stem cells in regenerative medicine. Front. Physiol. 2, 1–2 (2012).
2 Liras A. Future research and therapeutic applications of human stem cells: general,regulatory, and bioethical aspects. J. Transl.Med. 8, 131 (2010).
3 Liras A. Induced human pluripotent stem cells and advanced therapies:future perspectives for the treatment ofhaemophilia? Thromb. Res. 128(1), 8–13 (2011).
4 Vandendriessche T, Chuah MK. Clinical progress in gene therapy: sustained partial correction of the bleeding disorder in patients suffering from severe hemophilia B.
Hum. Gene Ther. 23(1), 4–6 (2012).
5 Liras A. Gene therapy for haemophilia: the end of a ‘royal pathology’ in the third millennium? Haemophilia 7(5), 441–445 (2001).
6 Li H, Haurigot V, Doyon Y et al. In vivo genome editing restores haemostasis in a mouse model of haemophilia. Nature 475(7355), 217–221 (2011).
7 Olmedillas S, García-Arranz M, Vega L,García-Gómez I, García-Olmo D, Liras A. Non-viral gene transfer to human adiposederived stem cells for FIX secretion. Presented
at: 4th National Congress of Spanish Society of Gene and Cellular Therapy. Zaragoza, Spain, 21–23 September 2011.
8 Nathwani AC, Tuddenham EG, Rangarajan S et al. Adenovirus-associated virus vectormediated gene transfer in hemophilia B. N. Engl. J. Med. 365(25), 2357–2365 (2011).
9 Mannucci PM, Tuddenham EG. The hemophilias – from royal genes to gene therapy. N. Engl. J. Med. 344(23),1773–1779 (2001).
10 Liras A, Olmedillas S. Gene therapy for haemophilia. Yes, but with non-viral vectors? Haemophilia 15(3), 811–816 (2009).
11 Ponder KP. Merry christmas for patients with hemophilia B. N. Engl. J. Med. 365(25), 2424–2425 (2011).
Website
101 European Medicines Agency. Advanced
therapies. www.ema.europa.eu/ema/index.jsp?curl=pages/special_topics/general/general_content_000504.jsp&mid=WC0b01ac058050f347