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

por Arechavala-Gomeza, Virginia, Gonzalez-Quereda, Lidia
Publicado 2021

Experimental Model Systems Used in the Preclinical Development of Nucleic Acid Therapeutics

por Zhou, Haiyan, Arechavala-Gomeza, Virginia, Garanto, Alejandro
Publicado 2023

Researcher's Perceptions on Publishing “Negative” Results and Open Access

por Echevarría, Lucía, Malerba, Alberto, Arechavala-Gomeza, Virginia
Publicado 2021

Splicing modulation therapy in the treatment of genetic diseases

por Arechavala-Gomeza, Virginia, Khoo, Bernard, Aartsma-Rus, Annemieke
Publicado 2014

The Biomarker Potential of miRNAs in Myotonic Dystrophy Type I

por Koehorst, Emma, Ballester-Lopez, Alfonsina, Arechavala-Gomeza, Virginia, Martínez-Piñeiro, Alicia, Nogales-Gadea, Gisela
Publicado 2020

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

por López-Martínez, Andrea, Soblechero-Martín, Patricia, de-la-Puente-Ovejero, Laura, Nogales-Gadea, Gisela, Arechavala-Gomeza, Virginia
Publicado 2020

Utrophin modulator drugs as potential therapies for Duchenne and Becker muscular dystrophies

por Soblechero‐Martín, Patricia, López‐Martínez, Andrea, de la Puente‐Ovejero, Laura, Vallejo‐Illarramendi, Ainara, Arechavala‐Gomeza, Virginia
Publicado 2021

Considerations in the Preclinical Assessment of the Safety of Antisense Oligonucleotides

por Goyenvalle, Aurélie, Jimenez-Mallebrera, Cecilia, van Roon, Willeke, Sewing, Sabine, Krieg, Arthur M., Arechavala-Gomeza, Virginia, Andersson, Patrik
Publicado 2023

A Duchenne Muscular Dystrophy Gene Hot Spot Mutation in Dystrophin-Deficient Cavalier King Charles Spaniels Is Amenable to Exon 51 Skipping

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

miR-106b is a novel target to promote muscle regeneration and restore satellite stem cell function in injured Duchenne dystrophic muscle

por Rodriguez-Outeiriño, Lara, Hernandez-Torres, Francisco, Ramirez de Acuña, Felicitas, Rastrojo, Alberto, Creus, Carlota, Carvajal, Alejandra, Salmeron, Luis, Montolio, Marisol, Soblechero-Martin, Patricia, Arechavala-Gomeza, Virginia, Franco, Diego, Aranega, Amelia Eva
Publicado 2022

Report of a TREAT-NMD/World Duchenne Organisation Meeting on Dystrophin Quantification Methodology

por Aartsma-Rus, Annemieke, Morgan, Jennifer, Lonkar, Pallavi, Neubert, Hendrik, Owens, Jane, Binks, Michael, Montolio, Marisol, Phadke, Rahul, Datson, Nicole, Van Deutekom, Judith, Morris, Glenn E., Rao, V. Ashutosh, Hoffman, Eric P., Muntoni, Francesco, Arechavala-Gomeza, Virginia
Publicado 2019

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

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

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

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

Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-...

por Cirak, Sebahattin, Arechavala-Gomeza, Virginia, Guglieri, Michela, Feng, Lucy, Torelli, Silvia, Anthony, Karen, Abbs, Stephen, Garralda, Maria Elena, Bourke, John, Wells, Dominic J, Dickson, George, Wood, Matthew JA, Wilton, Steve D, Straub, Volker, Kole, Ryszard, Shrewsbury, Stephen B, Sewry, Caroline, Morgan, Jennifer E, Bushby, Kate, Muntoni, Francesco
Publicado 2011

Dystrophin quantification: Biological and translational research implications

por Anthony, Karen, Arechavala-Gomeza, Virginia, Taylor, Laura E., Vulin, Adeline, Kaminoh, Yuuki, Torelli, Silvia, Feng, Lucy, Janghra, Narinder, Bonne, Gisèle, Beuvin, Maud, Barresi, Rita, Henderson, Matt, Laval, Steven, Lourbakos, Afrodite, Campion, Giles, Straub, Volker, Voit, Thomas, Sewry, Caroline A., Morgan, Jennifer E., Flanigan, Kevin M., Muntoni, Francesco
Publicado 2014

Delivery is key: lessons learnt from developing splice‐switching antisense therapies

por Godfrey, Caroline, Desviat, Lourdes R, Smedsrød, Bård, Piétri‐Rouxel, France, Denti, Michela A, Disterer, Petra, Lorain, Stéphanie, Nogales‐Gadea, Gisela, Sardone, Valentina, Anwar, Rayan, EL Andaloussi, Samir, Lehto, Taavi, Khoo, Bernard, Brolin, Camilla, van Roon‐Mom, Willeke MC, Goyenvalle, Aurélie, Aartsma‐Rus, Annemieke, Arechavala‐Gomeza, Virginia
Publicado 2017

Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-conce...

por Kinali, Maria, Arechavala-Gomeza, Virginia, Feng, Lucy, Cirak, Sebahattin, Hunt, David, Adkin, Carl, Guglieri, Michela, Ashton, Emma, Abbs, Stephen, Nihoyannopoulos, Petros, Garralda, Maria Elena, Rutherford, Mary, Mcculley, Caroline, Popplewell, Linda, Graham, Ian R, Dickson, George, Wood, Matthew JA, Wells, Dominic J, Wilton, Steve D, Kole, Ryszard, Straub, Volker, Bushby, Kate, Sewry, Caroline, Morgan, Jennifer E, Muntoni, Francesco
Publicado 2009

Joining European Scientific Forces to Face Pandemics

por Vasconcelos, M. Helena, Alcaro, Stefano, Arechavala-Gomeza, Virginia, Baumbach, Jan, Borges, Fernanda, Brevini, Tiziana A.L., Rivas, Javier De Las, Devaux, Yvan, Hozak, Pavel, Keinänen-Toivola, Minna M., Lattanzi, Giovanna, Mohr, Thomas, Murovska, Modra, Prusty, Bhupesh K., Quinlan, Roy A., Pérez-Sala, Dolores, Scheibenbogen, Carmen, Schmidt, Harald H.H.W., Silveira, Isabel, Tieri, Paolo, Tolios, Alexander, Riganti, Chiara
Publicado 2021

Delivery of oligonucleotide‐based therapeutics: challenges and opportunities

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

Progress and harmonization of gene editing to treat human diseases: Proceeding of COST Action CA21113 GenE-HumDi

por Cavazza, Alessia, Hendel, Ayal, Bak, Rasmus O., Rio, Paula, Güell, Marc, Lainšček, Duško, Arechavala-Gomeza, Virginia, Peng, Ling, Hapil, Fatma Zehra, Harvey, Joshua, Ortega, Francisco G., Gonzalez-Martinez, Coral, Lederer, Carsten W., Mikkelsen, Kasper, Gasiunas, Giedrius, Kalter, Nechama, Gonçalves, Manuel A.F.V., Petersen, Julie, Garanto, Alejandro, Montoliu, Lluis, Maresca, Marcello, Seemann, Stefan E., Gorodkin, Jan, Mazini, Loubna, Sanchez, Rosario, Rodriguez-Madoz, Juan R., Maldonado-Pérez, Noelia, Laura, Torella, Schmueck-Henneresse, Michael, Maccalli, Cristina, Grünewald, Julian, Carmona, Gloria, Kachamakova-Trojanowska, Neli, Miccio, Annarita, Martin, Francisco, Turchiano, Giandomenico, Cathomen, Toni, Luo, Yonglun, Tsai, Shengdar Q., Benabdellah, Karim
Publicado 2023