Adolfo López de Munain on Muck Rack

Adolfo López de Munain

As seen in: The American Journal of Human Genetics
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Articles

Enhanced muscle uptake of chemically optimized miR-23b antisense oligonucleotides as lead compounds for myotonic dystrophy type 1

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By Irene González-Martínez, Estefania Cerro-Herreros, Marc Carrascosa-Saez, Andrea García-Rey, Diego Piqueras-Losilla, Anna Colom-Rodrigo, Nerea Moreno, Mouli Chakraborty, Aline Huguet-Lachon, Anchel González-Barriga, Neia Naldaiz-Gastesi, Martxel Dehesa, Ana Díaz-Maqueda, Nuria Barquero, Miguel Varela , Ramon Eritja, Genevieve Gourdon, Arturo López-Castel, Manuel Pérez-Alonso, Beatriz Llamusi, Rubén Artero, Adolfo López de Munain
| The American Journal of Human Genetics
Keywords antisense oligonucleotides MBNL myotonic dystrophy oleic acid conjugate muscle uptake Introduction Myotonic dystrophy type 1 (DM1; MIM: 160900) is a rare genetic disorder with an overall prevalence of one in 3,000–8,000 individuals worldwide.1 DM1 is characterized by expansion of an unstable CTG microsatellite repeat in the 3′ untranslated region (UTR) of DM1 protein kinase (DMPK; MIM: 605377).
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