Открытый доступ Открытый доступ  Закрытый доступ Доступ платный или только для подписчиков

Вопросы разработки генотерапевтических лекарственных препаратов

Ekaterina Slobodkina, Pavel Makarevich, Andrey Dolinkin

Аннотация


Одним из перспективных направлений развития современной медицины является создание лекарственных препаратов для генной терапии. К настоящему времени в мире зарегистрировано порядка 10 генотерапевтических лекарственных средств. Ученые активно проводят доклинические и клинические исследования новых лекарственных препаратов, инновационный поиск новых подходов и методов повышения эффективности генной терапии. В обзоре представлены отдельные аспекты разработки генотерапевтических лекарственных средств: от рационального выбора и дизайна генетического вектора до особенностей производства и стратегии контроля качества готовой лекарственной формы.

Ключевые слова


генотерапевтические лекарственные препараты, невирусные векторы, плазмиды, стандартизация и контроль качества

Ссылки


Walsh C. E. Gene therapy progress and prospects: Gene therapy for the hemophilias / Gene Ther. 2003. V. 10. № 12. P. 999 – 1003.

Van Deutekom J. C. T., Van Ommen G. B. Advances in Duchenne muscular dystrophy gene therapy / Nat. Rev. Genet. 2003. V. 4. № 10. P. 774 – 783.

Ferrari S., Geddes D. M., Alton E. W. F. W. Barriers to and new approaches for gene therapy and gene delivery in cystic fibrosis / Adv. Drug Deliv. Rev. 2002. V. 54. № 11. P. 1373 – 1393.

O’Connor T., Crystal R. Genetic medicines: treatment strategies for hereditary disorders / Nat. Rev. Genet. 2006. V. 7. № 4. P. 1 – 76.

Bunnell B. A., Morgan R. A. Gene therapy for infectious diseases / Clin. Microbiol. Rev. 1998. V. 11. № 1. P. 42 – 56.

Dzau V. J. et al. Current perceptions of cardiovascular gene therapy / Am J Cardiol. 2003. Vol. 9149. № 3. P. 18 – 23.

Tuszynski M. H. Growth-factor gene therapy for neurodegenerative disorders / Lancet Neurol. 2002. V. 1. № 1. P. 51 – 57.

Vile R. G., Russell S. J., Lemoine N. R. Cancer gene therapy: hard lessons and new courses / Gene Ther. 2000. V. 7. № 1. P. 2 – 8.

Chen C. et al. Molecular characterization of adeno-associated viruses infecting children / J. Virol. 2005. V. 79. № 23. P. 14781 – 14792.

Li C. et al. Adeno-associated virus vectors: potential applications for cancer gene therapy / Cancer Gene Ther. 2005. V. 12. № 12. P. 913 – 925.

Cutroneo K. R. Gene therapy for tissue regeneration / J. Cell Biochem. 2003. V. 88. № 2. P. 418 – 425.

Wood K., Fry J. Gene therapy: potential applications in clinical transplantation. / Expert Rev. Mol. Med. 1999. P. 1 – 20.

Baum C. et al. Mutagenesis and oncogenesis by chromosomal insertion of gene transfer vectors / Hum. Gene Ther. 2006. V. 17. № 3. P. 253 – 263.

Bessis N., Garciacozar F. J., Boissier M. Immune responses to gene therapy vectors: influence on vector function and effector mechanisms / Gene Ther. 2004. 11. S10 – S17.

Waehler R., Russell S. J., Curiel D. T. Engineering targeted viral vectors for gene therapy / Nat. Rev. Genet. 2007. V. 8. № 8. P. 573 – 587.

Thomas C. E., Ehrhardt A., Kay M. A. Progress and problems with the use of viral vectors for gene therapy / Nat. Rev. Genet. 2003. V. 4. № 5. P. 346 – 358.

Bouard D., Cosset F. Viral vectors: from virology to transgene expression / Br. J. Pharmacol. 2009. V. 157. № 2. P. 153 – 165.

Ramamoorth M., Narvekar A. Non viral vectors in gene therapy — an overview / J. Clin. Diagn. Res. 2015. V. 9. № 1. P. 1 – 6.

San H. et al. Safety and short-term toxicity of a nov el cationic lipid formulation for human gene therapy / Hum. Gene Ther. 1993. V. 4. № 6. P. 781 – 788.

Morishita R. et al. Safety evaluation of clinical gene therapy using hepatocyte growth factor to treat peripheral arterial disease / Hypertension. 2004. V. 44. № 2. P. 203 – 209.

Dubensky T. J., Sauter S. Generation of retroviral packaging and producer cell lines for large-scale vector production with improved safety and titer / Meth. Mol. Med. 2003. V. 76. P. 309 – 330.

Mintzer M. A., Simanek E. E. Nonviral vectors for gene delivery / Chem. Rev. 2009. V. 109. № 2. P. 259 – 302.

Pack D. W. et al. Design and development of polymers for gene delivery / Nat. Rev. Drug Discov. 2005. V. 4. № 7. P. 581 – 593.

Wolff J. et al. Direct gene transfer into mouse muscle in vivo / Science. 1990. V. 247. № 4949. Pt 1. P. 1465 – 1468.

Kawabata K., Takakura Y., Hashida M. The fate of plasmid DNA after intravenous injection in mice: Involvement of scavenger receptors in its hepatic uptake / Pharm. Res. 1995. V. 12. № 6. P. 825 – 830.

Felgner J. H. et al. Enhanced gene delivery and mechanism studies with a novel series of cationic lipid formulations / J. Biol. Chem. 1994. V. 269. № 4. P. 2550 – 2561.

Gao X., Huang L. Cationic liposome-mediated gene transfer / Gene Ther. 1995. V. 35. № 3. P. 1027 – 1036.

Khalil I. A. et al. Uptake pathways and subsequent intracellular trafficking in nonviral gene delivery / Pharmacol. Rev. 2006. V. 58. № 1. P. 32 – 45.

Lechardeur D. et al. Metabolic instability of plasmid DNA in the cytosol: A potential barrier to gene transfer / Gene Ther. 1999. V. 6. № 4. P. 482 – 497.

Dowty M. E. et al. Plasmid DNA entry into postmitotic nuclei of primary rat myotubes / Proc. Natl. Acad. Sci. USA. 1995. V. 92. № 10. P. 4572 – 4576.

Luby-Phelps K. et al. Hindered diffusion of inert tracer particles in the cytoplasm of mouse 3T3 cells / Proc. Natl. Acad. Sci. USA. 1987. V. 84. № 14. P. 4910 – 4913.

Mislick K. A., Baldeschwieler J. D. Evidence for the role of proteoglycans in cation-mediated gene transfer / Proc. Natl. Acad. Sci. USA. 1996. V. 93. № 22. P. 12349 – 12354.

Erbacher P. et al. Putative role of chloroquine in gene transfer into a human hepatoma cell line by DNA / Lactosylated polylysine complexes / Exp. Cell Res. 1996. V. 225. № 1. P. 186 – 194.

Bonner W. M. Protein migration into nuclei. I. Frog oocyte nuclei in vivo accumulate microinjected histones, allow entry to small proteins, and exclude large proteins / J. Cell Biol. 1975. V. 64. № 2. P. 421 – 430.

Featherstone C., Darby M. K., Gerace L. A Monoclonal antibody against the nuclear pore complex inhibits nucleocytoplasmic transport of protein and RNA in vivo / J. Cell Biol. 1988. V. 107. № 4. P. 1289 – 1297.

Lukacs G. L. et al. Size-dependent DNA mobility in cytoplasm and nucleus / J. Biol. Chem. 2000. V. 275. № 3. P. 1625 – 1629.

Sebesty?n M. et al. DNA vector chemistry: the covalent attachment of signal peptides to plasmid DNA / Nat. Biotechnol. 1998. V. 16. № 1. P. 80 – 85.

Miller A. M., Dean D. A. Tissue-specific and transcription factor-mediated nuclear entry of DNA / Adv. Drug Deliv. Rev. Elsevier B. V., 2009. V. 61. № 7 – 8. P. 603 – 613.

Wilke M. et al. Efficacy of a peptide-based gene delivery system depends on mitotic activity / Gene Ther. 1996. V. 3. № 12. P. 1133 – 1142.

Brunner S. et al. Cell cycle dependence of gene transfer by lipoplex, polyplex and recombinant adenovirus / Gene Ther. 2000. V. 7. № 5. P. 401 – 407.

EMA. Directive 2001/83/EC of the European Parliament and of the Council [Electronic resource]. 2001. URL: http://bit.ly/2OSmGEK.

FDA. Guidance for Industry: Gene Therapy Clinical Trials — Observing Subjects for Delayed Adverse Events [Electronic resource]. 2006. URL: http:// bit.ly/2PZjZO3.

?1047? Gene Therapy Products / US Pharmacopeia 38 NF. 2015.

SFDA. Points to Consider for Human Gene Therapy and Product Quality Control State Food and Drug Administration of China / BioPharm Int. 2004. P. 1 – 4.

Миронов А. и др. Состояние и перспективы развития генотерапии в России / Вестн. Росздравнадзора. 2011. № 4. С. 56 – 60.

Супотницкий М. В. и др. Особенности доклинических исследований на этапе разработки невирусных векторных конструкций, предназначенных для целей генной терапии / Ведом. НЦ экспертизы средств мед. примен. 2014. № 3. С. 30 – 38.

EMA. Guideline on the quality, non-clinical and clinical aspects of gene therapy medicinal products. 2015. V. 44. March. P. 1 – 42.

EMEA. Summary of product characteristics — Glybera. 2012.

Vatsan R. S. Summary Basis for Regulatory Action — IMLYGIC. 2015.

Liebert M. A. Current status of Gendicine in China: Recombinant human Ad-p53 agent for treatment of cancers / Gene Med. 2005. V. 16. № 9. P. 1016 – 1027.

Rivera V. M. et al. Long-term pharmacologically regulated expression of erythropoietin in primates following AAV-mediated gene transfer / Blood. 2005. V. 105. № 4. P. 1424 – 1430.

Wang L. et al. Sustained correction of disease in naive and AAV2-pretreated hemophilia B dogs: AAV2/8-mediated, liver-directed gene therapy / Blood. 2005. V. 105. № 8. P. 3079 – 3086.

Xiao X., Li J. Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector / J. Virol. 1996. V. 70. № 11. P. 8098 – 8108.

Jiang H. et al. Evidence of multiyear factor IX expression by AAV-mediated gene transfer to skeletal muscle in an individual with severe hemophilia B / Mol. Ther. 2006. V. 14. № 3. P. 452 – 455.

Walsh G. Pharmaceutical Biotechnology. The Atrium, Southern Gate, Chichester, West Sussex PO19 8SQ, England: John Wiley & Sons Ltd, 2007.

Будыльская Т. В. и др. Фармацевтическая разработка ГЛФ биофармацевтических препаратов для генной терапии / Разраб. и рег. лек. средств. 2016. Т. 1. № 14.С. 74 – 85.


Полный текст: PDF

Ссылки

  • Ссылки не определены.


** ** ** ** ** **

ISSN: 2073-8099

** ** ** ** ** **

Подписаться на наши издания Вы можете через почтовые каталоги Объединенный каталог «Пресса России» «Урал Пресс», «Ивис»«Прессинформ» и «Профиздат».

 

Наши партнеры:

iIPhEB - Международная выставка и форум по фармацевтике и биотехнологиям, 2–4 апреля 2024

Семинар R&D для R&D, 12–13 апреля 2024