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Gene Therapy in the Developing Countries

Emengaha, Festus Chidi


Nnodim Johnkennedy


Okorie Hope


Amah Henry


Edward Ukamaka

Emengaha, Festus Chidi 1 Nnodim Johnkennedy 1 ,
Okorie Hope 1 Amah Henry 1 Edward Ukamaka 1 

  1. Department of Medical Laboratory Science, Faculty of Health Science, Imo State University Owerri, Imo State, Nigeria 1

Pages: 80-92

DOI: 10.18488/journal.9/2015.2.5/

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 Gene therapy is treatment directed to curing genetic disease by introducing normal genes into the patients in order to overcome the effects of abnormal genes using technique of genetic engineering. The most radical approach involves applying this at the early stage in the embrayo, so that the new gene would be incorporated into the germ cells and hence will be inheritable.  This approach is not considered to be safe either safe or ethical because the consequences may affect the descendants of the patients. Therefore, this review throws more light on gene therapy, its application and consequences in the developing countries.  
Contribution/ Originality
This study contributes in the existing literature, in new gene therapy approach that repairs errors in messenger RNA derived from defective genes. It is now theoretically possible to cure all genetic diseases. It is now imperative that developing countries should embrace gene therapy technology.




  1. D. Pezzoli, "We still have a long way to go to effectively deliver genes," J. Appl. Biomater Funct Mater, vol. 10, pp. 82-91, 2012.
  2. C. Sheridan, "Gene therapy finds its niche," Nature Biotechnology, vol. 29, pp. 121-128, 2011.
  3. A. M. Maguire, F. Simonelli, and F. Simonelli, "Safety and efficacy of gene transfer for leber's congenital amaurosis," New England Journal of Medicine, vol. 358, pp. 2240-2248, 2008.
  4. G. Kolata, After setbacks, small successes for gene therapy vol. 3. New York Times, 2009.
  5. M. Herper, "Gene therapy's big comeback forbes magazine," Forbes Magazine, vol. 8, p. 23, 2014.
  6. J. Gallagher, "Gene therapy: Glybera approved by European commission," Science, vol. 13, p. 66, 2012.
  7. P. A. Lewitt, A. R. Rezai, M. A. Leehey, S. G. Ojemann, A. W. Flaherty, E. N. Eskandar, S. K. Kostyk, K. Thomas, A. Sarkar, M. S. Siddiqui, S. B. Tatter, J. M. Schwalb, K. L. Poston, J. M. Henderson, R. M. Kurlan, I. H. Richard, L. Van Meter, C. V. Sapan, M. J. During, M. G. Kaplitt, and A. Feigin, "AAV2-GAD gene therapy for advanced parkinson's disease: A double-blind, sham-surgery controlled, randomised trial," The Lancet Neurology, vol. 10, pp. 309-319, 2011.
  8. L. Vannucci, "Viral vectors: A look back and ahead on gene transfer technology," New Microbiol, vol. 36, pp. 1-22, 2013.
  9. D. Urnov Fyodor, J. Rebar Edward, M. Holmes, C., H. Zhang, and D. Steve Gregory Philip, "Genome editing with engineered zinc finger nucleases," Nature Reviews Genetics, vol. 11, pp. 636-646, 2010.
  10. K. Hanna, Germline gene transfer vol. 2: National Human Genome Research Institute, 2006.
  11. R. M. Blaese, K. W. Culver, A. D. Miller, C. S. Carter, T. Fleisher, M. Clerici, G. Shearer, L. Chang, Y. Chiang,
  12. P. Tolstoshev, J. J. Greenblatt, S. A. Rosenberg, H. Klein, M. Berger, C. A. Mullen, W. J. Ramsey, L. Muul, R.
  13. Morgan, and W. F. Anderson, "T lymphocyte-directed gene therapy for ADA- SCID: Initial trial results after 4 years," Science, vol. 270, pp. 475-480, 1995.
  14. F. Wilson Jennifer, "Murine gene therapy corrects symptoms of sickle cell disease," The Scientist-Magazine of the Life Sciences, vol. 7, pp. 16-22, 2002.
  15. St. Jude Children's Research Hospital, "Gene therapy corrects sickle cell disease in laboratory study," Science Daily, vol. 4, pp. 11-12, 2008.
  16. A. Abbott, "Gene therapy. Italians first to use stem cells," Nature, vol. 356, pp. 465-199, 1992.
  17. M. Cavazzana-Calvo, A. Thrasher, and F. Mavilio, "The future of gene therapy," Nature, vol. 427, pp. 779-781, 2004.
  18. R. Stein, "First patient treated in stem cell study: Washington post," vol. 2, pp. 14-15, 2010.
  19. G. Stolberg Sheryl, "Gene therapy ordered halted at university. New York Times," vol. 2, pp. 18-21, 2000.
  20. A. Ananthaswamy, "Undercover genes slip into the brain," New Scientist, vol. 3, pp. 11-14, 2003.
  21. B. Holmes, "Gene therapy may switch off huntington's," New Scientist, vol. 3, pp. 7-9, 2003.
  22. M. G. Ott, M. Schmidt, K. Schwarzwaelder, S. Stein, U. Siler, U. Koehl, H. Glimm, K. Kiihlcke, A. Schilz, H. Kunkel, S. Naundorf, A. Brinkmann, A. Deichmann, M. Fischer, C. Ball, I. Pilz, C. Dunbar, Y. Du, N. A. Jenkins, N. G. Copeland, U. Luthi, M. Hassan, A. J. Thrasher, D. Hoelzer, C. Von Kalle, R. Seger, and M. Grez, "Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1," Nature Medicine, vol. 12, pp. 401-409, 2006.
  23. B. D. Brown, M. A. Venneri, A. Zingale, L. S. Sergi, and L. Naldini, "Endogenous micro RNA regulation suppresses transgene expression in hematopoietic lineages and enables stable gene transfer," Nature Medicine, vol. 12, pp. 585-591, 2006.
  24. R. A. Morgan, M. E. Dudley, J. R. Wunderlich, M. S. Hughes, J. C. Yang, R. M. Sherry, R. E. Royal, S. L. Topalian, U. S. Kammula, N. P. Restifo, Z. Zheng, A. Nahvi, C. R. De Vries, L. J. Rogers-Freezer, S. A. Mavroukakis, and S. A. Rosenberg, "Cancer regression in patients after transfer of genetically engineered lymphocytes," Science, vol. 314, pp. 126-129, 2006.
  25. B. L. Levine, L. M. Humeau, J. Boyer, R. R. MacGregor, T. Rebello, X. Lu, G. K. Binder, V. Slepushkin, F. Lemiale, J. R. Mascola, F. D. Bushman, B. Dropulic, and C. H. June, "Gene transfer in humans using a conditionally replicating lentiviral vector," Science, vol. 314, pp. 121-122, 2006.
  26. E. Dolgin, "Colour blindness corrected by gene therapy," Nature, vol. 2, pp. 66-69, 2009.
  27. N. Cartier and P. Aubourg, "Hematopoietic stem cell transplantation and hematopoietic stem cell gene therapy in x-linked adrenoleukodystrophy," Brain Pathology, vol. 20, pp. 857-862, 2009.
  28. A. Komaromy, J. Alexander, J. Rowlan, M. Garcia, V. Chiodo, A. Kaya, J. Tanaka, G. Acland, W. Hauswirth, and G. D. Aguirre, "Gene therapy rescues cone function in congenital achromatopsia," Human Molecular Genetics, vol. 19, pp. 2581-2593, 2010.
  29. M. Cavazzana-Calvo, E. Payen, O. Negre, G. Wang, K. Hehir, F. Fusil, I. Down, M. Denaro, T. Brady, K. Westerman, R. Cavallesco, B. Gillet-Legrand, L. Caccavelli, R. Sgarra, L. Maouche-Chretien, F. O. Bernaudin, R. Girot, R. Dorazio, G. J. Mulder, A. Polack, A. Bank, J. Soulier, J. R. M. Larghero, N. Kabbara, B. Dalle, B. Gourmel, G. R. Socie, S. Chretien, N. Cartier, and P. Aubourg, "Transfusion independence and HMGA2 activation after gene therapy of human p-thalassaemia," Nature, vol. 467, p. 318, 2010.
  30. R. Galanello and R. Origa, "Beta-thalassemia," Orphanet Journal of Rare Diseases, vol. 5, p. 11, 2010.
  31. Leboulch and Philippe, "Five year outcome of lentiviral gene therapy for human beta-thalassemia, lessons and prospects thalassemia reports," in Proceedings of the 3rd Pan-European Conference on Haemoglobinopathies and Rare Anaemias, 2013.
  32. K. Beals Jacquelyn, "Gene therapy frees beta-thalassemia patient from transfusions for 2+ years," Science, vol. 43, p. 313, 2010.
  33. B-Thalassemia, "Major with autologous CD34+ hematopoietic progenitor cells transduced with TNS9.3.55 a lentiviral vector encoding the normal human 6-globin gene cancer center," Clinical Trials, vol. 4, p. 113, 2012.
  34. T. Rosenberg, "The man who had HIV and now does not New York magazine," vol. 2, p. 76, 2011.
  35. H. Ledford, "Cell therapy fights leukaemia," Nature, vol. 37, pp. 472-477, 2011.
  36. Z. J. Yang, Y. R. Zhang, B. Chen, S. L. Zhang, E. Z. Jia, L. S. Wang, T. B. Zhu, C. I. Li, H. Wang, I. Huang, K. J. Cao, W. Z. Ma, B. Wu, L. S. Wang, and C. T. Wu, "Phase I clinical trial on intracoronary administration of Ad-hHGF treating severe coronary artery disease," Molecular Biology Reports, vol. 36, pp. 1323-1329, 2008.
  37. W. Hahn, W. B. Pyun, D. S. Kim, W. S. Yoo, S. D. Lee, J. H. Won, G. J. Shin, J. M. Kim, and S. Kim, "Enhanced cardioprotective effects by coexpression of two isoforms of hepatocyte growth factor from naked plasmid DNA in a rat ischemic heart disease model," The Journal of Gene Medicine, vol. 13, pp. 549-555, 2011.
  38. A. Pollack, "European agency backs approval of a gene therapy New York times," vol. 2, p. 16, 2012.
  39. A. Coghlan, "Gene therapy cures leukaemia in eight days," The New Scientist, vol. 9, pp. 15-23, 2013.
  40. Bosely and Sarah, "Pioneering gene therapy trials offer hope for heart patients the Guardian," vol. 18, p. 23, 2013.
  41. A. Aiuti, L. Biasco, S. Scaramuzza, F. Ferrua, M. P. Cicalese, C. Baricordi, F. Dionisio, A. Calabria, S. Giannelli, M. C. Castiello, M. Bosticardo, C. Evangelic, A. Assanelli, M. Casiraghi, S. Di Nunzio, L. Callegaro, C. Benati, P. Rizzardi, D. Pellin, C. Di Serio, M. Schmidt, C. Von Kalle, J. Gardner, N. Mehta, V. Neduva, D. J. Dow, A. Galy, R. Miniero, A. Finocchi, and A. Metin, "Lentiviral hematopoietic stem cell gene therapy in patients with wiskott-aldrich syndrome," Science, vol. 341, p. 1233151, 2013.
  42. L. Geddes, "Bubble kid' success puts gene therapy back on track," The New Scientist, vol. 4, pp. 42-43, 2013.
  43. R. E. MacLaren, M. Groppe, A. R. Barnard, C. L. Cottriall, T. Tolmachova, L. Seymour, K. R. Clark, M. J. During, F. P. M. Cremers, G. C. M. Black, A. J. Lotery, S. M. Downes, A. R. Webster, and M. C. Seabra, "Retinal gene therapy in patients with choroideremia: Initial findings from a phase 1/2 clinical trial," The Lancet Neurology, vol. 7, pp. 62-64 2014.
  44. P. Tebas, D. Stein, W. W. Tang, I. Frank, S. Q. Wang, G. Lee, S. K. Spratt, R. T. Surosky, M. A. Giedlin, G. Nichol, M. C. Holmes, P. D. Gregory, D. G. Ando, M. Kalos, R. G. Collman, G. Binder-Scholl, G. Plesa, W. T. Hwang, B. L. Levine, and C. H. June, "Gene editing ofCCRSin autologous CD4 T cells of persons infected with HIV," New England Journal of Medicine, vol. 370, p. 901, 2014.
  45. G. Dvorsky, "Scientists create genetically modified cells that protect against HIV," Biotechnology, vol. 2, pp. 6-14, 2014.
  46. F. Baylis and S. R. Jason, "The inevitability of genetic enhancement technologies," Bioethics, vol. 18, pp. 1-26, 2004.
  47. J. Evans, Playing God?: Human genetic engineering and the rationalization of public bioethical debate vol. 2: University of Chicago Press, 2002.
  48. N. B. Woods, V. Bottero, M. Schmidt, C. Von Kalle, and I. M. Verma, "Gene therapy: Therapeutic gene causing lymphoma," Nature, vol. 440, p. 1123, 2006.
  49. A. J. Thrasher, H. B. Gaspar, C. Baum, U. Modlich, A. Schambach, F. Candotti, M. Otsu, B. Sorrentino, L. Scobie, E. Cameron, K. Blyth, J. Neil, S. H. B. Abina, M. Cavazzana-Calvo, and A. Fischer, "Gene therapy. X-SCID transgene leukaemogenicity," Nature, vol. 443, pp. E5-E6, 2006.
  50. S. Young, "Genome surgery MIT technology " Review, vol. 3, pp. 17-19, 2014.
  51. J. Pap Schedel, G. Pap Moller Ladner, and R. E. Gayl Gumcamp, "Gene therapy in osteoarthritis," Joint Bone Spine, vol. 5, p. 38, 2000.


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