{"ok":true,"nota":{"id":"ed4a2e6d24b7775b","slug":"un-farmaco-experimental-reduce-en-ratones-las-proteinas-toxicas-que-ca-ed4a2e","titulo":"Un fármaco experimental reduce en ratones las proteínas tóxicas que causan la enfermedad de Huntington","resumen":"Investigadores probaron VO659, un oligonucleótido antisentido que apunta a la secuencia genética defectuosa. Midieron cuánto baja la producción de varias proteínas dañinas asociadas a la enfermedad.","fuente":"biorxiv","fuenteNombre":"bioRxiv · preprints","color":"#be2628","autor":"Röttgering, B., Weij, R., Bijl, S., Verheul, R., Testerink, J., Toonen, L., Bragg, R., Carroll, J. B., Beekman, C., Dat","url":"https://www.biorxiv.org/content/10.64898/2026.10.16.758452v1?rss=1","imagen":"/hoy/portada/ed4a2e6d24b7775b","tema":"salud","subtemas":["enfermedad de huntington","terapia genética","oligonucleótidos","proteínas tóxicas","neurología"],"entidades":["VO659","bioRxiv"],"formato":"dato","entretenimiento":false,"pubTs":"2026-10-22T00:00:00.000Z","indexTs":"2026-10-22T16:38:44.280Z","hash":"5e3d6a5af89a917211888dee1bf6cca126f21b1abe4d168563bf5f0220c506b9","tsa":{"time":"Oct 22 16:39:43 2026 GMT","provider":"freetsa.org","sobre":"sello"},"stats":{"views":0,"likes":0,"clicks":0,"dwell":0},"tipo":"normal","registro":"hallazgo","porQueImporta":"El tratamiento redujo varias proteínas tóxicas a la vez, y el efecto fue mayor en los casos con repeticiones genéticas más largas, que son las que suelen causar la forma más grave. Para familias argentinas que conviven con esta enfermedad hereditaria, es una señal de que se busca atacar la causa y no sólo los síntomas. Aun así, son resultados preliminares en laboratorio y falta mucho para saber si funcionan y son seguros en personas.","quienFirma":"Vico Therapeutics","confianza":"preprint","consulta":null},"sello":{"hash":"5e3d6a5af89a917211888dee1bf6cca126f21b1abe4d168563bf5f0220c506b9","sello":"7fa0e7c07045c0f19dff610383518da00c0533c1d82ba0cd7c693a1fc2f4b3a2","beacon":{"drand":{"cadena":"8990e7a9aaed2ffed73dbd7092123d6f289930540d7651336225dc172e51b2ce","ronda":6575204,"aleatorio":"ac0f4a6ce509202540c9641abb3a70f7f06eeb36f68a34e1bc3e430369a775da","tiempo":"2026-10-22T16:39:00.000Z"},"nist":{"cadena":2,"pulso":1994801,"salida":"123765DCC46130497358D1E6393BF76FC82D205E2F260DEF171C4887B733C1209F31A193DDD877AB50C2E9F79D0A125A26712AD900F817275AC0E40E5C1C39BC","tiempo":"2026-10-22T16:38:00.000Z"},"noAntes":"2026-10-22T16:39:00.000Z"},"verificacion":{"drand":true,"nist":true},"tsa":{"time":"Oct 22 16:39:43 2026 GMT","provider":"freetsa.org","sobre":"sello"},"original":{"titulo":"VO659, a CAG repeat-targeting antisense oligonucleotide, lowers multiple neurotoxic mutant huntingtin proteins in Huntington's disease","descripcion":"Huntington's disease (HD) is caused by a CAG repeat expansion in the huntingtin (HTT) gene, encoding an elongated polyglutamine stretch in the corresponding mutant HTT (mHTT) protein. HD pathogenesis is driven by multiple neurotoxic mHTT protein species, including HTT1a and repeat-associated non-AUG translation (RAN) proteins, which increase in expression as mutant CAG repeats lengthen due to somatic expansion. VO659 is an investigational CAG repeat-targeting antisense oligonucleotide (ASO) that lowers mHTT protein levels by blocking translation. Here, we show that VO659 allele-preferentially lowers levels of multiple neurotoxic mHTT protein species in a CAG repeat length-dependent manner, r","pubDate":"2026-10-22","url":"https://www.biorxiv.org/content/10.64898/2026.10.16.758452v1?rss=1"},"indexado":"2026-10-22T16:38:44.280Z"}}