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Colegio de Medicina Interna de México.

2006, Number 5

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Med Int Mex 2006; 22 (5)

Genical therapy

Vera LOL

Language: Spanish
References: 85
Page: 422-438
PDF size: 337.66 Kb.


ABSTRACT

Gene therapy (GT) is a therapeutic strategy characterized by introduction of genetic material into a patient with the goal of correcting cellular deficiencies expressed at phenotype level and to cure hereditary and acquired diseases. GT constitutes a great hope for patients who suffer some genetic disturbance or any other disease, since an altered gene can be replaced by a normal one: The transfer of genes is made through vectors, which are systems used in the process of transferring one exogen gene into a cell, facilitating the entrance and intracellular bioavailability of it. Vectors can be classified as follows 1) viral as the retrovirus, adenovirus, adeno-associated virus and others. 2) Non- viral as the particle-mediated up take, direct injection of DNA, cationic liposomes, transfer trough receptors, among others. The main applications of GT are: 1) Inherited disorders: hemoglobinopathies, hereditary immunodeficiency, etc., 2) cancer, 3) autoimmune diseases: Rheumatoid arthritis, systemic lupus erythematosus, and 4) others like infections (AIDS), ischemic cardiopathy. Many of the genetic and acquired disease are potentially treatable by GT. However, this treatment has had great difficulties. Some studies phase I and II with diverse viral and non-viral vectors have been performed; but yet these are many problems that need to be solved like the efficiency and the host immune response as well as its safety. GT is still a hope for the future treatment of many diseases. It is expected that with the development of technology and new vectors, the efficiency and safety of GT viral improve.


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