Hace dos años, estos investigadores informaron del primer éxito logrado con terapia génica en dos niños afectados por esta grave enfermedad, conocida popularmente como síndrome del "niño burbuja".
En la mitad de los casos está causada por un defecto genético en el cromosoma X, en un gen esencial para el desarrollo de ciertos linfocitos cuyo papel es combatir los virus y bacterias.
Los autores explican que han conseguido tratar la inmunodeficiencia en 4 de 5 niños extrayendo su médula ósea, corrigiendo el defecto genético en las células inmunes inmaduras y reimplantando las células corregidas. A los 4 meses, los niños ya disponían de linfocitos maduros, y la respuesta de los linfocitos T era normal a los 2 años de la intervención.
Los niños tenían entre 1 y 11 meses cuando fueron sometidos al tratamiento. Los dos pacientes originales han sido seguidos durante 2,5 años, mientras que otros 3 tratados con éxito lo han sido entre 1,5 y 2 años.
Webs Relacionadas
Hôpital Necker-Enfants Malades
http://www.necker.fr/
New England Journal of Medicine
http://content.nejm.org/
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