Hemofilia: la terapia génica sería la solución

Un experimento de terapia génica realizado en perros abre el camino a una nueva esperanza de tratamiento para los hemofílicos.

La hemofilia es una enfermedad hereditaria grave que provoca la incapacidad de la sangre para coagularse. Cuando una persona con hemofilia comienza a sangrar, el flujo no puede detenerse o es muy difícil. Las consecuencias pueden ser complicadas con hemorragias graves en caso de lesión y a veces hemorragias espontáneas, especialmente en las articulaciones (llamadas hemartrosis).

Existen dos formas de hemofilia: la hemofilia A es la más común. Afecta a un niño de cada 5.000 nacimientos y se caracteriza por una deficiencia de la proteína que ayuda a la coagulación de la sangre (factor VIII de coagulación).

La hemofilia no se cura y sólo puede controlarse con tratamientos que inyectan agentes coagulantes. Sin embargo, acaba de surgir una esperanza de tratamiento con terapia génica, tras un ensayo terapéutico realizado en perros con hemofilia A (porque los perros reproducen la enfermedad de la misma manera que los humanos).

Un equipo de investigadores de la Universidad de Wisconsin (Estados Unidos) y del Hospital Xavier Arnozan de Pessac (Gironda) publicó los resultados en la revista Nature Communications.

El equipo franco-americano utilizó un virus para transportar el factor de coagulación a las plaquetas a través de la médula espinal. Los animales y la evolución de la enfermedad fueron seguidos durante más de dos años. Al final de este período de vigilancia, dos de los tres perros ya no mostraban síntomas de hemofilia.

Para continuar su investigación, los científicos presentaron una solicitud de ensayo clínico en los Estados Unidos. Esto podría hacerse en los próximos dos años.

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