Un futuro fármaco inyectable para el tratamiento del síndrome de Down

Cada año nacen en Chile unos 500 niños que padecen Síndrome de Down, un trastorno genético caracterizado por el deterioro cognitivo en grados variables. Afortunadamente, las últimas investigaciones en torno a la enfermedad se acercan cada vez más a una potencial cura. En julio pasado, expertos de la Universidad de Harvard demostraron por primera vez en la historia que es posible neutralizar de forma natural elcromosoma 21, responsable de esta discapacidad que provoca dificultades en el desarrollo cognitivo, rasgos faciales distintivos y otros problemas de salud.
Ahora, un grupo de científicos de laUniversidad Johns Hopkins de Medicinaexperimentan con un nuevo fármaco inyectable que podría normalizar el desarrollo cognitivo de los recién nacidos diagnosticados.
Según informa el Science Translational Medicine Journal, hasta el momento el compuesto ha sido sólo testeado en pruebas con ratones que fueron científicamente modificados para tener condiciones equivalentes al Síndrome de Down en humanos.
Al cumplir un día de vida, se les inyectó el nuevo compuesto fabricado a partir de una molécula conocida en inglés como sonichedgehogpathwayagonist. Los resultados sorprendieron a los expertos que lograron normalizar el crecimiento del cerebelo con una sola inyección.
Si bien este hallazgo puede significar un avance notable que podría abrir las puertas para una potencial cura, Roger Reeves, miembro del equipo investigador, se mostró cauteloso ante la posibilidad de aplicarlo en humanos en un futuro cercano.

Fuente: Universia Chile

Autor: Universia Chile

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