NUEVA NOTICIA DE GENOTIPIA. Una terapia génica ralentiza un 75% la progresión de la enfermedad de Huntington

octubre 13, 2025/en Científicas, Sin categorizar

Una terapia génica experimental llamada AMT-130 ha logrado frenar de forma significativa la progresión de la enfermedad de Huntington, según un estudio de la University College London y la empresa uniQure. Con una sola dosis administrada directamente en el cerebro, los pacientes tratados mostraron una reducción del 75% en la progresión clínica y mejoras en funciones motoras y cognitivas.

El tratamiento introduce material genético terapéutico en las neuronas para reducir la proteína huntingtina mutante, responsable de la enfermedad. Además, se observó una disminución de los biomarcadores de daño neuronal y buena tolerancia general al procedimiento.

uniQure planea solicitar en 2026 la aprobación acelerada ante la FDA, lo que podría convertir a AMT-130 en el primer tratamiento capaz de modificar el curso de esta enfermedad neurodegenerativa y abrir nuevas vías para otras patologías genéticas del cerebro.

Fuente: genotipia

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