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Med. Regenerativa

19/05/2017

Células sanguíneas cultivadas en el Laboratorio

Por Nicolás Jouve, Catedrático Emérito de Genética. Presidente de CiViCa

Se acaban de publicar en Nature (17 de Mayo) unos trabajos de dos equipos de científicos de una gran trascendencia para el futuro tratamiento de enfermedades sanguíneas. Lo que se consigue en estas investigaciones es la identificación de unos factores de transcripción (reguladores de la expresión genética) y también factores ambientales que permiten reprogramar células humanas y de ratón para convertirlas en células troncales (células madre) progenitoras del sistema hematopoyético, a su vez generadoras de las células sanguíneas. En ambos trabajos, las células generadas producen exitosamente células sanguíneas tras ser implantadas en la médula ósea de ratones de laboratorio.

18/05/2017

Aprobado un ensayo clínico de “edición génica” en células humanas de hígado, in vivo

Por Nicolás Jouve, Catedrático Emérito de Genética y Presidente de CiViCa. Noticia de la que se puede encontrar más información en el artículo de Abby Olena publicado en The Scientist el 18 de Mayo de 2017 (ilustración de Medical Graphics).

La compañía biotecnológica Sangamo Therapeutics utilizará una tecnología de edición génica, anterior a la CRISPR-Cas9, que utiliza una enzima nucleasa de “dedos de zinc” para eliminar y corregir regiones de genes implicados en varias enfermedades: hemofilia B, síndrome de Hurler  y síndrome de Hunter,  en hígados humanos.

18/05/2017

Utilización de células reprogramadas (iPSCs) por primera vez en pacientes de degeneración de retina

Por Nicolás Jouve, Catedrático Emérito de Genética. Presidente de CiViCa.

El pasado 28 de Marzo, se comunicó en  Nature News  la aplicación por primera vez a un hombre de 60 años de edad, una terapia para la degeneración macular con células inducidas pluripotentes iPSCs de acuerdo al protocolo desarrollado en Japón por Takahashi. Con anterioridad el equipo de Takahashi comunicó el éxito de la reprogramación hacia células iPSC de la retina derivadas del propio paciente (Fotografía: Wet age-related macular degeneration FLICKR, COMMUNITY EYE HEALTH)

07/03/2017

Posible primer tratamiento para una grave enfermedad rara

Publicado en Madri+d, fuente UAM, el 6 de Marzo de 2017

PMM2-CDG es el subtipo más común de los 'defectos congénitos de la glicosilación', un grupo de enfermedades metabólicas hereditarias en los que el proceso de adición de un glúcido a otra molécula (proteínas/lípidos) es deficiente, lo que causa un grave malfuncionamiento en órganos y sistemas vitales. Se conocen más de 100 defectos genéticos diferentes que afectan a la glicosilación de proteínas, siendo la PMM2-CDG el defecto más común que afecta a menos de mil personas en el mundo y para las que no existe actualmente ningún tratamiento.