El ADN de un hombre vivo directamente modificado

ADN modificado en una persona viva

Los científicos californianos han podido modificar directamente el ADN de un hombre vivo utilizando la terapia génietca de infusión. Algo que puede empezar a cambiar los tratamientos de enfermedades.

Un importante paso adelante en la terapia génetica. Los médicos del Hospital Oakland en California, EE.UU., han editado el ADN de un estadounidense añadiendo un nuevo gen. El paciente de 44 años padece la enfermedad de Hunter, una rara enfermedad genética que daña gradualmente las células del cuerpo.

Los pacientes con síndrome de Hunter sufren de una deficiencia de una enzima que descompone los mucopolisacáridos, las cadenas de moléculas de azúcar utilizadas para construir los tejidos conectivos (tejidos que sostienen y llenan el cuerpo).

Esta deficiencia conduce a síntomas de una enfermedad degenerativa como problemas de audición, defectos óseos y articulares, y dificultades cardíacas y respiratorias. La enfermedad de Hunter no se puede curar completamente, pero se puede controlar con terapias de reemplazo enzimático.

En la actualidad, los pacientes que reciben tratamientos regulares siguen sufriendo daños progresivos en el corazón, los huesos y los pulmones.

Por lo tanto, los médicos californianos han probado un nuevo enfoque de la terapia génetica realizada directamente en el ADN de un hombre vivo. Inyectaron miles de millones de copias de un gen corrector y tijeras moleculares en el cuerpo del paciente para cortar el ADN en un lugar específico.

Esta técnica de edición genética se dirige a secciones del ADN que necesitan ser modificadas o reparadas para reemplazarlas.

El procedimiento es excepcional porque la inyección se realiza generalmente en células previamente recolectadas antes de ser reintroducidas en el paciente. En el caso de esta persona, no se tomaron muestras. Una infusión de dos a tres horas fue suficiente para insertar los genes correctivos en el cuerpo.

Un proceso irreversible

Es demasiado pronto para saber si este experimento es concluyente, los primeros resultados serán visibles en uno a tres meses, según los científicos. La apuesta es arriesgada porque una vez que los genes han sido inyectados, ya no es posible volver atrás.

“Cuando se coloca aleatoriamente un trozo de ADN, a veces funciona bien, a veces no importa y a veces causa daño”, dice Hank Greely, un bioeticista de la Universidad de Stanford. Los investigadores siguen siendo optimistas. Si este tratamiento innovador funciona, marcará un hito importante en el campo de la terapia génetica.

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