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Tuesday, March 20, 2007

Womb, gene therapy

http://www.youtube.com/watch?v=vfeAJxtb-F8


Many people or families know and have suffered in flesh and blood the devastating effects of a genetic illness that affects severely anyone of their newborns without previous warning. Illnesses like Hemophilia, Syndrome of Down, Cystic fibrosis, Sickle cell anemia and many metabolic illnesses that attack fetuses with permanent (for a lifetime) defects, having the therapy -mostly- palliative effects, as long as the damage (as in most of the cases) already has been done. The future could change in about 5 years, if the experiments of genetic therapy (substitution -in uterus- of abnormal genes, for other normal ones driven to the fetal cells by carrier virus), are effective in human. The therapy has been successful in fetuses of hemophilic mice where the illness was reverted ad integrum. Now, British scientists get ready to carry out the same experiment in animals but big : fetuses of sheep and primates, for finally to be carried out in human. According to Simón Waddington (College London) and Charles Coutelle (Imperial College), 2 obstacles conspire against a genetic successful therapy: 1) the immune rejection mounted by the host body to the implanted genes, fact that one waits to overcome working with fetuses where the immune answer is weak and 2) the development in some fetuses (years but late), of cancer, effect that is hoped to revert with carefully planned and watched studies. As it is known USA and France some years ago suspended their tests because a boy tried with genetic therapy in fetal state, developed cancer at 3 years of age.



http://www.youtube.com/watch?v=EfXK50Bxod8


Terapia genética intra utero

Muchas personas o familias conocen y han sufrido en carne propia los devastadores efectos de una enfermedad genética, que sin aviso previo afecta severamente a cualquiera de sus recien nacidos. Enfermedades como la Hemofilia, el Síndrome de Down, la Fibrosis Quistica, la Anemia falciforme y otras enfermedades metabólicas agreden a los afectados con defectos permanentes (para toda la vida), teniendo mayormente la terapia efectos paliativos, en tanto el daño ya está hecho (como lo es en la mayoria de los casos). El futuro podria cambiar dentro de unos 5 años, si los experimentos de terapia genética (reemplazo de genes anormales in utero, por otros normales conducidos a las células fetales por virus), resultan efectivos en humanos. La terapia ya es exitosa al menos en fetos de ratones hemofilicos donde la enfermedad logró ser revertida ad integrum. Ahora, cientificos británicos se preparan para realizar el mismo experimento en animales mas grandes : :fetos de ovejas y primates, para finalmente ser realizados en humanos. Según Simon Waddington (College London) y Charles Coutelle (Imperial collage), 2 obstáculos conspiran contra una terapia genética exitosa : 1) el rechazo inmune del soma del feto afectado a los genes implantados, hecho que se espera remontar trabajando con fetos donde la respuesta inmune es debil y 2) el desarrollo en algunos fetos (años mas tarde) de cancer, efecto que se espera revertir con estudios cuidadosamente planificados y observados. Como se sabe hace algunos años atrás USA y Francia suspendieron sus ensayos porque un niño tratado con terapia genética en estado fetal, desarrolló cancer a los 3 años de edad.




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