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Colegio de Medicina Interna de México.

2006, Número 5

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Med Int Mex 2006; 22 (5)


Terapia génica

Vera LOL

Idioma: Español
Referencias bibliográficas: 85
Paginas: 422-438
Archivo PDF: 337.66 Kb.


RESUMEN

La terapia génica es una estrategia terapéutica que implica la introducción de material genético en los pacientes, con la finalidad de corregir las deficiencias celulares expresadas en el fenotipo y sanar enfermedades hereditarias o adquiridas. La terapia génica es de gran esperanza para los pacientes que padecen alguna alteración genética u otra enfermedad ya que se puede sustituir el gen alterado por otro normal. La transferencia de genes se realiza por medio de vectores; estos son sistemas que se utilizan en el proceso de transferencia de un gen exógeno a la célula facilitando la entrada y la biodisponibilidad intracelular del mismo. Los vectores se dividen en 1) virales: retrovirus, adenovirus, virus adenoasociados y otros; 2) no virales: bombardeo de partículas, inyección directa de ADN, liposomas catiónicos, transferencia mediante receptores, entre otros. Las principales aplicaciones de la terapia génica son: 1) enfermedades hereditarias: hemoglobinopatías, inmunodeficiencias, etc; 2) cáncer; 3) enfermedades autoinmunitarias: artritis reumatoide, lupus eritematoso sistémico; y 4) otras como las infecciones: SIDA, las cardiopatías isquémicas y otras. Muchas de las enfermedades genéticas y adquiridas son potencialmente tratables con terapia génica; sin embargo, esta terapéutica ha tenido grandes dificultades. Se han realizado estudios de fase I y II con diversos vectores virales y no virales, pero hay problemas por resolver, como la eficacia y la respuesta inmunitaria del huésped, además de su inocuidad. La terapia génica sigue siendo la esperanza para el tratamiento futuro de varias enfermedades. Se espera que el progreso de la tecnología y los nuevos vectores mejorarán la eficacia e inocuidad de esta terapia.


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