Special Issue “Genetic Advances in Neuromuscular Disorders: From Gene Identification to Gene Therapy”

by Virginia Arechavala-Gomeza, Lidia Gonzalez-Quereda
Published 2021-02-01

The Biomarker Potential of miRNAs in Myotonic Dystrophy Type I

by Emma Koehorst, Alfonsina Ballester-Lopez, Virginia Arechavala-Gomeza, Alicia Martínez-Piñeiro, Gisela Nogales-Gadea
Published 2020-12-01

An Overview of Alternative Splicing Defects Implicated in Myotonic Dystrophy Type I

by Andrea López-Martínez, Patricia Soblechero-Martín, Laura de-la-Puente-Ovejero, Gisela Nogales-Gadea, Virginia Arechavala-Gomeza
Published 2020-09-01

A duchenne muscular dystrophy gene hot spot mutation in dystrophin-deficient cavalier king charles spaniels is amenable to exon 51 skipping.

by Gemma L Walmsley, Virginia Arechavala-Gomeza, Marta Fernandez-Fuente, Margaret M Burke, Nicole Nagel, Angela Holder, Rachael Stanley, Kate Chandler, Stanley L Marks, Francesco Muntoni, G Diane Shelton, Richard J Piercy
Published 2010-01-01

Duchenne muscular dystrophy cell culture models created by CRISPR/Cas9 gene editing and their application in drug screening 

by Patricia Soblechero-Martín, Edurne Albiasu-Arteta, Aina Anton-Martinez, Laura de la Puente-Ovejero, Iker Garcia-Jimenez, Gabriela González-Iglesias, Irene Larrañaga-Aiestaran, Andrea López-Martínez, Javier Poyatos-García, Estíbaliz Ruiz-Del-Yerro, Federico Gonzalez, Virginia Arechavala-Gomeza
Published 2021-09-01

A multicenter comparison of quantification methods for antisense oligonucleotide-induced DMD exon 51 skipping in Duchenne muscular dystrophy cell cultures.

by Monika Hiller, Maria Sofia Falzarano, Iker Garcia-Jimenez, Valentina Sardone, Ruurd C Verheul, Linda Popplewell, Karen Anthony, Estibaliz Ruiz-Del-Yerro, Hana Osman, Jelle J Goeman, Kamel Mamchaoui, George Dickson, Alessandra Ferlini, Francesco Muntoni, Annemieke Aartsma-Rus, Virginia Arechavala-Gomeza, Nicole A Datson, Pietro Spitali
Published 2018-01-01

Delivery of oligonucleotide‐based therapeutics: challenges and opportunities

by Suzan M Hammond, Annemieke Aartsma‐Rus, Sandra Alves, Sven E Borgos, Ronald A M Buijsen, Rob W J Collin, Giuseppina Covello, Michela A Denti, Lourdes R Desviat, Lucía Echevarría, Camilla Foged, Gisela Gaina, Alejandro Garanto, Aurelie T Goyenvalle, Magdalena Guzowska, Irina Holodnuka, David R Jones, Sabine Krause, Taavi Lehto, Marisol Montolio, Willeke Van Roon‐Mom, Virginia Arechavala‐Gomeza
Published 2021-04-01