Алипоген типарвовек: долгая дорога к оценке отношения пользы и риска генотерапевтического препарата
Резюме
Об авторах
Р. Н. АляутдинРоссия
Б. К. Романов
Россия
А. П. Переверзев
Россия
А. О. Чикало
Россия
Н. Д. Бунятян
Россия
В. А. Меркулов
Россия
А. Н. Миронов
Россия
Список литературы
1. Nehoff H., Parayath N.N., Domanovitch L., Taurin S., Greish K. Nanomedicine for drug targeting: strategies beyond the enhanced permeability and retention effect. J. of Nanomed. 2014; (9): 2539-55.
2. Аляутдин Р.Н., Кройтер Й., Харкевич Д.А. Доставка лекарственных препаратов в мозг с помощью наночастиц. Экспериментальная и клиническая фармакология 2003; (66): 65-68.
3. Bryant L.M., Christopher D.M., Giles AR., et al. Lessons learned from the clinical development and market authorization of Glybera. Hum Gene Ther Clin Dev. 2013; (24): 55-64.
4. Nierman M.C., Rip J., Twick J. Gene therapy for genetic lipoprotein lipase deficiency: from promise to practice. The Netherlands J. of Med. 2005; (63): 14-22.
5. Kimura W., Mossner J. Role of hypertriglyceridemia in the pathogenesis of experimental acute pancreatitis in rats. Int J Pancreatol. 1996; (20): 177-184.
6. Weber C.K., Adler G. From Acinar Cell Damage to Systemic Inflammatory Response: Current Concepts in Pancreatitis. Pancreatology 2001; (1): 356-62.
7. Bach A.C., Babayan V.K. Medium-chain triglycerides: an update. Am J Clin Nutr. 1982; (36): 950-62.
8. Durrington P. Dyslipidaemia. Lancet 2003; (362): 717-31.
9. Eckel R.H. Lipoprotein lipase. A multifunctional enzyme relevant to common metabolic diseases. N Engl J Med. 1989; (320): 1060-68.
10. Effects of lovaza on lipoprotein composition and function in mild hypertriglyceridemia (ELLF). Available from: http://clinicaltrials.gov/ct2/show/NCT00901979.
11. Van Dam M., Stalenhoef A.F., Wittekoek J. Efficacy of concentrated n-3 Fatty acids in hypertriglyceridaemia: a comparison with gemfibrozil. Clin Drug Invest. 2001; (21): 175-81.
12. Shyam D., Berns K.I. Gene therapy using adeno-associated virus vector. Clin Microbiol Review 2008; (43): 583-93.
13. Супотницкий М.В. Генотерапевтические векторные системы на основе вирусов. Биопрепараты 2011; (3): 15-26.
14. Gruntman A.M., Bish L.T., Mueller C., et al. Gene transfer in skeletal and cardiac muscle using recombinant adeno-associated virus. Curr Prot Microbiol 2013; (14): 3-12.
15. Fisher K.J., Jooss K., Alston J. Recombinant adeno-associated for muscle directed gene therapy. Nat Med. 1997; (3): 306-31.
16. Rip J., Nierman M.C., Jukema J.W., et al. Lipoprotein lipase S447X - a naturellyoc-curring gain-of-function. Arter Thromb Vasc Biol. 2006; (6): 1236-45.
17. Wang D., Zhong L., Abu Nahid M., et al. The potential of adeno-associated viral vectors for gene delivery to muscle tissue. Expert Opin Drug Deliv. 2014; (11): 345-64.
18. Burnett J., Hooper A.J. Alipogene tiparvovec, an adeno-associated virus encoding the Ser447X variant of the human lipoprotein lipase gene for the treanment of patients with lipoprotein lipase deficiency. Curr Opinion in Mol Ther. 2009; (11): 681-91.
19. Ross C.J., Liu G., Kuenvenhoven J.A., et al. Complete rescue of lipoprotein lipase-deficient mice by somatic gene transfer of the naturally occurring LPL beneficial mutation. Arter Thromb Vasc Biol. 2005; (25): 2143-50.
20. Ross C.J., Twisk J., Bakker A.C., et al. Correction of feline lipoprotein lipase deficiency with adeno-associated virus serotype 1-vediated gene transfer of the lipoprotein lipase S447X beneficial mutation. Hum Gene Ther 2006; (17): 487-99.
21. Ferreira V., Petry H., Salmon F. Immune Responses to AAV-Vectors, the Glybera Example from Bench to Bedside. Front Immunol. 2014; (5): 82-85.
22. Buning H. Gene therapy enters the pharma market: the short story of a long journey. EMBO Mol Med. 2013; (5): 1-3.
Рецензия
Для цитирования:
Аляутдин Р.Н., Романов Б.К., Переверзев А.П., Чикало А.О., Бунятян Н.Д., Меркулов В.А., Миронов А.Н. Алипоген типарвовек: долгая дорога к оценке отношения пользы и риска генотерапевтического препарата. Ведомости Научного центра экспертизы средств медицинского применения. 2015;(1):31-34.
For citation:
Alyautdin R.N., Romanov B.K., Pereverzev A.P., Chikalo A.O., Bunyatyan N.D., Merkulov V.A., Mironov A.N. Alipogene tiparvovec: a long journey of risk-benefit ratio assessment of gene therapy products. The Bulletin of the Scientific Centre for Expert Evaluation of Medicinal Products. 2015;(1):31-34. (In Russ.)