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Gene therapy is the use of DNA as a pharmaceutical agent to treat disease. It derives its name from the idea that DNA can be used to supplement or alter genes within an individual's cells as a therapy to treat disease. The most common form of gene therapy involves using DNA that encodes a functional, therapeutic gene to replace a mutated gene. Other forms involve directly correcting a mutation, or using DNA that encodes a therapeutic protein drug (rather than a natural human gene) to provide treatment. In gene therapy, DNA that encodes a therapeutic protein is packaged within a "vector", which is used to get the DNA inside cells within the body. Once inside, the DNA becomes expressed by the cell machinery, resulting in the production of therapeutic protein, which in turn treats the patient's disease.

Gene therapy was first conceptualized in 1972, with the authors urging caution before commencing gene therapy studies in humans. The first FDA-approved gene therapy experiment in theUnited States occurred in 1990, when Ashanti DeSilva was treated for ADA-SCID.[1] Since then, over 1,700 clinical trials have been conducted using a number of techniques for gene therapy.[2]

Although early clinical failures led many to dismiss gene therapy as over-hyped, clinical successes since 2006 have bolstered new optimism in the promise of gene therapy. These include successful treatment of patients with the retinal disease Leber's congenital amaurosis,[3][4][5][6] X-linked SCID,[7] ADASCID,[8][9] adrenoleukodystrophy,[10] chronic lymphocytic leukemia (CLL),[11] acute lymphocytic leukemia (ALL),[12] multiple myeloma,[13] haemophilia[9] and Parkinson's disease.[14] These recent clinical successes have led to a renewed interest in gene therapy, with several articles in scientific and popular publications calling for continued investment in the field.[15][16]

In 2012, Glybera became the first gene therapy treatment to be approved for clinical use in either Europe or the United States after its endorsement by the European Commission.[17][18]


Gene therapy may be classified into the two following types:

(1) Somatic gene therapy (2) Germ line gene therapy

Somatic gene therapy

In somatic gene therapy, the therapeutic genes are transferred into the somatic cells (non sex-cells), or body, of a patient. Any modifications and effects will be restricted to the individual patient only, and will not be inherited by the patient's offspring or later generations. Somatic gene therapy represents the mainstream line of current basic and clinical research, where the therapeutic DNA transgene (either integrated in the genome or as an external episome or plasmid) is used to treat a disease in an individual.

Germ line gene therapy

In germ line gene therapy, germ cells (sperm or eggs), are modified by the introduction of functional genes, which are integrated into their genomes. Germ cells will combine to form a zygote which will divide to produce all the other cells in an organism and therefore if a germ cell is genetically modified then all the cells in the organism will contain the modified gene. This would allow the therapy to be heritable and passed on to later generations. Although this should, in theory, be highly effective in counteracting genetic disorders and hereditary diseases, some jurisdictions, including Australia, Canada, Germany, Israel, Switzerland, and the Netherlands[21] prohibit this for application in human beings, at least for the present, for technical and ethical reasons, including insufficient knowledge about possible risks to future generations [21] and higher risk than somatic gene therapy (e.g. using non-integrative vectors).[

1. 2. 3. ^ Jump up to: Sheridan, C. (2011). "Gene therapy finds its niche". Nature Biotechnology 29 (2): 121 128. doi:10.1038/nbt.1769.PMID 21301435. edit a b ^ Jump up to: J. Gene Med. Gene Therapy Clinical Trials Database. a b ^ Jump up to: Maguire, A. M.; Simonelli, F.; Pierce, E. A.; Pugh Jr, E. N.; Mingozzi, F.; Bennicelli, J.; Banfi, S.; Marshall, K. A.; Testa, F.; Surace, E. M.; Rossi, S.; Lyubarsky, A.; Arruda, V. R.; Konkle, B.; Stone, E.; Sun, J.; Jacobs, J.; Dell'Osso, L.; Hertle, R.; Ma, J. X.; Redmond, T. M.; Zhu, X.; Hauck, B.; Zelenaia, O.; Shindler, K. S.; Maguire, M. G.; Wright, J. F.; Volpe, N. J.; McDonnell, J. W.; Auricchio, A. (2008). "Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis". New England Journal of Medicine 358 (21): 22402248. doi:10.1056/NEJMoa0802315. PMC 2829748. PMID 18441370. edit
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^ Jump up to: Simonelli, F.; Maguire, A. M.; Testa, F.; Pierce, E. A.; Mingozzi, F.; Bennicelli, J. L.; Rossi, S.; Marshall, K.; Banfi, S.; Surace, E. M.; Sun, J.; Redmond, T. M.; Zhu, X.; Shindler, K. S.; Ying, G. S.; Ziviello, C.; Acerra, C.; Wright, J. F.; McDonnell, J. W.; High, K. A.; Bennett, J.; Auricchio, A. (2009). "Gene Therapy for Leber's Congenital Amaurosis is Safe and Effective Through 1.5 Years After Vector Administration". Molecular Therapy 18 (3): 643 650. doi:10.1038/mt.2009.277.PMC 2839440. PMID 19953081. edit a b 5. ^ Jump up to: Cideciyan, A. V.; Hauswirth, W. W.; Aleman, T. S.; Kaushal, S.; Schwartz, S. B.; Boye, S. L.; Windsor, E. A. M.; Conlon, T. J.; Sumaroka, A.; Roman, A. J.; Byrne, B. J.; Jacobson, S. G. (2009). "Vision 1 Year after Gene Therapy for Leber's Congenital Amaurosis". New England Journal of Medicine 361 (7): 725727. doi:10.1056/NEJMc0903652. PMC 2847775.PMID 19675341. edit a b 6. ^ Jump up to: Bainbridge, J. W. B.; Smith, A. J.; Barker, S. S.; Robbie, S.; Henderson, R.; Balaggan, K.; Viswanathan, A.; Holder, G. E.; Stockman, A.; Tyler, N.; Petersen-Jones, S.; Bhattacharya, S. S.; Thrasher, A. J.; Fitzke, F. W.; Carter, B. J.; Rubin, G. S.; Moore, A. T.; Ali, R. R. (2008). "Effect of Gene Therapy on Visual Function in Leber's Congenital Amaurosis". New England Journal of Medicine 358 (21): 2231 2239. doi:10.1056/NEJMoa0802268. PMID 18441371. edit 7. Jump up^ Fischer, A.; Hacein-Bey-Abina, S.; Cavazzana-Calvo, M. (2010). "20 years of gene therapy for SCID". Nature Immunology 11(6): 457460. doi:10.1038/ni0610-457. PMID 20485269. edit 8. Jump up^ Ferrua, F.; Brigida, I.; Aiuti, A. (2010). "Update on gene therapy for adenosine deaminasedeficient severe combined immunodeficiency". Current Opinion in Allergy and Clinical Immunology 10 (6): 551556. doi:10.1097/ACI.0b013e32833fea85.PMID 20966749. edit a b c d 9. ^ Jump up to: Geddes, Linda (30 October 2013) 'Bubble kid' success puts gene therapy back on track' The New Scientist, Retrieved 2 November 2013 10. Jump up^ Cartier, N.; Aubourg, P. (2009). "Hematopoietic Stem Cell Transplantation and Hematopoietic Stem Cell Gene Therapy in X-Linked Adrenoleukodystrophy". Brain Pathology 20 (4): 857 862. doi:10.1111/j.1750-3639.2010.00394.x.PMID 20626747. edit a b 11. ^ Jump up to: Ledford, H. (2011). "Cell therapy fights leukaemia". Nature. doi:10.1038/news.2011.472. edit a b 12. ^ Jump up to: Coghlan, Andy (26 March 2013) Gene therapy cures leukaemia in eight days The New Scientist, Retrieved 15 April 2013

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