Progress in gene therapy


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Abstract

Recent progress in gene therapy, current status of investigations in this area of experimental medicine are reviewed. Much attention is given to gene-therapeutic approaches the efficacy of which is proved in clinical trials.

About the authors

Dina Viktorovna Glazkova

Email: glazkova@pcr.ru

Elena Vladimirovna Bogoslovskaya

Email: lenabo@pcr.ru

German Aleksandrovich Shipulin

Email: german@pcr.ru

Valentin Ivanovich Pokrovskiy

Email: info@pcr.ru

D V Glazkova

Central Research Institute of Epidemiology, Moscow

Central Research Institute of Epidemiology, Moscow

E V Bogoslovskaya

Central Research Institute of Epidemiology, Moscow

Central Research Institute of Epidemiology, Moscow

G A Shipulin

Central Research Institute of Epidemiology, Moscow

Central Research Institute of Epidemiology, Moscow

V I Pokrovsky

Central Research Institute of Epidemiology, Moscow

Central Research Institute of Epidemiology, Moscow

References

  1. Breakthrough of the year. The runners-up. Science 2009; 326 (5960): 1600-1607.
  2. Heilbronn R., Weger S. Viral vectors for gene transfer: current status of gene therapeutics. Handb. Exp. Pharmacol. 2010; 197: 143-170.
  3. Viola J. R., El-Andaloussi S., Oprea I. I., Smith С. I. Non-viral nanovectors for gene delivery: factors that govern successful therapeutics. Expert Opin. Drug Deliv. 2010; 7 (6): 721-735.
  4. Blaese R. M., Culver K. W., Miller A. D. et al. Т lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years. Science 1995; 270 (5235): 475-480.
  5. Somia N., Verma I. M. Gene therapy: trials and tribulations. Nat. Rev. Genet. 2000; 1 (2): 91-99.
  6. Hacein-Bey-Abina S., Garrigue A., Wang G. P. et al. Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. J. Сlin. Invest. 2008; 118 (9): 3132- 3142.
  7. Rosenberg S. A., Aebersold P., Cornetta К. et al. Gene transfer into humans - immunotherapy of patients with advanced melanoma, using tumor-infiltrating lymphocytes modified by retroviral gene transduction. N. Engl. J. Med. 1990; 323 (9): 570-578.
  8. Aiuti A., Roncarolo M. G. Ten years of gene therapy for primary immune deficiencies. In: Hematol. Am. Soc. Hematol. Educ. Program. 2009. 682-689.
  9. Aiuti A., Cattaneo F., Galimberti S. et al. Gene therapy for immunodeficiency due to adenosine deaminase deficiency. N. Engl. J. Med. 2009; 360 (5): 447-548.
  10. Hacein-Bey-Abina S., Hauer J., Lim A. et al. Efficacy of gene therapy for X-linked severe combined immunodeficiency. N. Engl. J. Med. 2010; 363 (4): 355-364.
  11. Gaspar H. B., Parsley K. L., Howe S. et al. Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector. Lancet 2004; 364 (9452): 2181- 2187.
  12. European Community clinical trials database EudraCT No.: 2007-000684-16: http://eudract.emea.europa.en.
  13. Zhang F., Thornhill S. I., Howe S. J. et al. Lentiviral vectors containing an enhancer-less ubiquitously acting chromatin opening element (UCOE) provide highly reproducible and stable transgene expression in hematopoietic cells. Blood 2007; 110 (5): 1448-1457.
  14. Thornhill S. I., Schambach A., Howe S. J. et al. Self-inactivating gammaretroviral vectors for gene therapy of X-linked severe combined immunodeficiency. Mol. Ther. 2008; 16 (3): 590-598.
  15. Kang E. M., Malech H. L. Advances in treatment for chronic granulomatous disease. Immunol. Res. 2009; 43 (l-3): 77-84.
  16. Ott M. G., Schmidt M., Schwarzwaelder K. et al. Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Nat. Med. 2006; 12 (4): 401-409.
  17. Kang E. M., Choi U., Theobald N. et al. Retrovirus gene therapy for X-linked chronic granulomatous disease can achieve stable long-term correction of oxidase activity in peripheral blood neutrophils. Blood 2010; 115 (4): 783-791.
  18. Boztug K., Schmidt M., Schwarzer A. et al. Correction of Wiskott-Aldrich syndrome by hematopoietic stem cell gene therapy. In: XVIII Annual congress of the European Society of Gene and Cell Therapy. (ESGCT) Odober 22-25, 2010. Milan, Italy: 49.
  19. Pawliuk R., Westerman K. A., Fabry M. E. et al. Correction of sickle cell disease in transgenic mouse models by gene therapy. Science 2001; 294 (5550): 2368-2371.
  20. Sadelain M., Lisowski L., Samakoglu S. et al. Progress toward the genetic treatment of the beta-thalassemias. Ann. H. Y. Acad. Sci. 2005; 1054: 78-91.
  21. Kaiser J. Gene therapy: Beta-thalassemia treatment succeeds, with a caveat. Science 2009; 326 (5959): 1468-1469.
  22. Semmler A., Köhler W., Jung H. H. et al. Therapy of X-linked adrenoleukodystrophy. Expert Rev. Neurother. 2008; 8 (9): 1367-1379.
  23. Cartier N., Hacein-Bey-Abina S., Bartholomae C. C. et al. Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Science 2009; 326 (5954): 818-823.
  24. Sharma A., Tandon M., Bangari D. S., Mittal S. K. Adenoviral vector-based strategies for cancer therapy. Curr. Drug Ther. 2009; 4 (2): 117-138.
  25. Sherr C. J., McCormick F. The RB and p53 pathways in cancer. Cancer Cell 2002; 2 (2): 103-112.
  26. Roth J. A. Adenovirus p53 gene therapy. Expert Opin. Biol. Ther. 2006; 6 (1): 55-61.
  27. Peng Z. Current status of gendicine in China: recombinant human Ad-p53 agent for treatment of cancers. Hum Gene Ther. 2005; 1 6 (9): 1016-1027.
  28. INGN 201: Ad-p53, Ad5CMV-p53, adenoviral p53, p53 gene therapy-introgen, RPR/INGN 201. Drugsin R. D. 2007; 8 (3): 176-187.
  29. Shimada H., Matsubara H., Shiratori T. et al. Phase I/II adenoviral p53 gene therapy for chemoradiation resistant advanced esophageal squamous cell carcinoma. Cancer Sci. 2006; 97 (6): 554-561.
  30. Yu W., Fang H. Clinical trials with oncolytic adenovirus in China. Curr. Cancer Drug Targets 2007; 7 (2):141-148.
  31. Khuri F. R., Nemunaitis J., Ganly I., et al. A controlled trial of intratumoral ONYX-015, a selectively-replicating adenovirus, in combination with cisplatin and 5-fluorouracil in patients with recurrent head and neck cancer, Nat. Med. 2000; 6 (8): 879-885.
  32. Aghi M., Martuza R. L. Oncolytic viral therapies - the clinical experience. Oncogene 2005; 24 (52): 7802-7816.
  33. Aghi M., Kramm C. M., Chou T. C. et al. Synergistic anticancer effects of ganciclovir/thymidine kinase and 5-fiuorocytosine/cytosine deaminase gene therapies. J. Natl. Cancer Inst. 1998; 90 (5): 370-380.
  34. Freytag S. O., Movsas B., Aref I. et al. Phase I trial of replication-competent adenovirus-mediated suicide gene therapy combined with IMRT for prostate cancer. Mol. Ther. 2007; 15 (5): 1016-1023.
  35. Freytag S. O., Stricker H., Peabody J. et al. Five-year follow-up of trial of replication-competent adenovirus-mediated suicide gene therapy for treatment of prostate cancer. Mol. Ther. 2007; 15 (3): 636-642.
  36. Brenner M. K., Okur F. V. Overview of gene therapy clinical progress including cancer treatment with gene-modified Т cells. In: Hematol. Am. Soc. Hematol. Educ. Program. 2009: 675-681.
  37. Westwood J. A., Kershaw M. H. Genetic redirection of Т cells for cancer therapy. J. Leukoc. Biol. 2010; 87 (5): 791-803.
  38. Morgan R. A., Dudley M. E., Wunderlich J. R. et al. Cancer regression in patients after transfer of genetically engineered lymphocytes. Science 2006; 314 (5796): 126-129.
  39. Johnson L. A., Morgan R. A., Dudley M. E. et al. Gene therapy with human and mouse T-cell receptors mediates cancer regression and targets normal tissues expressing cognate antigen. Blood 2009; 114 (3): 535-546.
  40. Park J. R., Digiusto D. L., Slovak M. et al. Adoptive transfer of chimeric antigen receptor re-directed cytolytic Т lymphocyte clones in patients with neuroblastoma. Mol. Ther. 2007; 15 (4): 825-833.
  41. Pule M. A., Savoldo B., Myers G. D. et al. Virus-specific Т cells engineered to coexpress tumor-specific receptors: persistence and antitumor activity in individuals with neuroblastoma. Nat. Med. 2008; 14 (11): 1264-1270.
  42. Mondino A., Dardalhon V., Michelini R. H. et al. Redirecting the immune response: role of adoptive Т cell therapy. Hum. Gene Ther. 2010; 21 (5): 533-541.
  43. Koido S., Нага Е., Homma S. et al. Cancer vaccine by fusions of dendritic and cancer cells. Clin. Dev. Immunol. 2009; 2009:657369: 1-13.
  44. Bollard C. M., Gottschalk S., Leen A. M. et al. Complete responses of relapsed lymphoma following genetic modification of tumor-antigen presenting cells and T-lymphocyte transfer. Blood 2007; 110 (8): 2838-2845.
  45. Straathof К. С., Bollard C. M., Popat U. et al. Treatment of nasopharyngeal carcinoma with Epstein - Barr virus-specific Т lymphocytes. Blood. 2005; 105 (5): 1898-1904.
  46. Leen A. M., Christin A., Myers G. D. et al. Cytotoxic Т lymphocyte therapy with donor Т cells prevents and treats adenovirus and Epstein - Barr virus infections after haploidentical and matched unrelated stem cell transplantation. Blood 2009; 114 (19): 4283-4292.
  47. Gerdemann U., Christin A. S., Vera J. F. et al. Nucleofection of DCs to generate Multivirus-specific Т cells for prevention or treatment of viral infections in the immunocompromised host. Mol. Tier. 2009; 17 (9): 1616-1625.
  48. Van Tendeloo V. F., Van de Velde A., Van Driessche A. et al. Induction of complete and molecular remissions in acute myeloid leukemia by Wilms' tumor 1 antigen-targeted dendritic cell vaccination. Proc. Natl. Acad. Sci. USA. 2010; 107 (31): 13824-13829.
  49. Von Laer D., Baum C., Protzer U. Antiviral gene therapy. Handb. Exp. Pharmacol. 2009; 189: 265-297.
  50. Rossi J. J., June C. H., Kohn D. B. Genetic therapies against HIV. Nat. Biotechnol. 2007; 25 (12): 1444-1454.
  51. Anderson J., Li M. J., Palmer B. et al. Safety and efficacy of a lentiviral vector containing three anti-HIV genes - CCR5 ribozyme, tat-rev siRNA, and TAR decoy - in SCID-hu mouse-derived Т cells. Mol. Ther. 2007; 15 (6): 1182-1188.
  52. http://www. natap.org/2009/HIV/012709_01.htm
  53. Gupta R., Tongers J., Losordo D. W. Human studies of angiogenic gene therapy. Circ. Res. 2009; 105 (8): 724-736.
  54. Бокерия Л. А., Аракелян B. C., Еремеева М. В. и др. Опыт лечения хронической ишемии нижних конечностей с помощью стимуляторов ангиогенеза. Клеточ. технол. в биол. и мед. 2007; 3: 159-164.
  55. Jazwa A., Jozkowicz A., Dulak J. New vectors and strategies for cardiovasculargene therapy. Curr. Gene Ther. 2007; 7 (l): 7- 23.
  56. Roy K., Stein L., Kaushal S. Ocular gene therapy: an evaluation of recombinant adeno-associated virus-mediated gene therapy interventions for the treatment of ocular disease. Hum. Gene Ther. 2010; 21 (8): 915-927.
  57. Bainbridge J. W., Ali R. R. Success in sight: The eyes have it! Ocular gene therapy trials for LCA look promising. Gene Ther. 2008; 15 (17): 1191-1192.
  58. Maguire A. M., High K. A., Auricchio A. et al. Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial. Lancet 2009; 374 (9701): 1597-1605.
  59. Mancuso K., Hauswirth W. W., Li Q. et al. Gene therapy for red-green colour blindness in adult primates. Nature 2009; 461 (7265): 784-787.
  60. Feng L. R., Maguire-Zeiss K. A. Gene therapy in Parkinson's disease: rationale and current status. CNS Drugs 2010; 24 (3): 177-192.
  61. Kaplitt M. G., Feigin A., Tang C. et al. Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial. Lancet 2007; 369 (9579): 2097-2105.
  62. Muramatsu S., Fujimoto K., Kato S. et al. A phase I study of aromatic L-amino acid decarboxylase gene therapy for Parkinson's disease. Mol. Ther. 2010; 18 (9): 1731-1735.
  63. Jarraya B., Boulet S., Ralph G. S. et al. Dopamine gene therapy for Parkinson's disease in a nonhuman primate without associated dyskinesia. Sci. Transl. Med. 2009; l (2): 2-4.
  64. Zhong L., Li B., Mah C. S. et al. Next generation of adeno-associated virus 2 vectors: point mutations in tyrosines lead to high-efficiency transduction at lower doses. Proc. Natl. Acad. Sci. USA 2008; 105 (22): 7827-7832.
  65. Asokan A., Conway J. C., Phillips J. L. et al. Reengineeringa receptor footprint of adeno-associated virus enables selective and systemic gene transfer to muscle. Nat. Biotechnol. 2010; 28 (1): 79-82.
  66. Marquez R. T., McCaffrey A. P. Advances in microRNAs: implications for gene therapists. Hum. Gene Ther. 2008; 19 (1): 27-38.
  67. Klapper J. A., Thomasian A. A., Smith D. M. et al. Single-pass, closed-system rapid expansion of lymphocyte cultures for adoptive cell therapy. J. Immunol. Meth. 2009; 345 (1-2): 90-99.
  68. Hackett P. B., Largaespada D. A., Cooper L. J. A transposon and transposase system for human application. Mol. Ther. 2010; 18 (4): 674-683.

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