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Delivery is key: lessons learnt from developing splice-switching antisense therapies.
Godfrey C, Desviat LR, Smedsrød B, Piétri-Rouxel F, Denti MA, Disterer P, Lorain S, Nogales-Gadea G, Sardone V, Anwar R, El Andaloussi S, Lehto T, Khoo B, Brolin C, van Roon-Mom WM, Goyenvalle A, Aartsma-Rus A, Arechavala-Gomeza V. Godfrey C, et al. EMBO Mol Med. 2017 May;9(5):545-557. doi: 10.15252/emmm.201607199. EMBO Mol Med. 2017. PMID: 28289078 Free PMC article. Review.
COST Actions: fostering collaborative research for rare diseases.
Desviat LR, Mallebrera CJ, Vallejo-Illarramendi A, Mayán MD, Nogales-Gadea G, Arechavala-Gomeza V. Desviat LR, et al. Lancet Neurol. 2019 Nov;18(11):989-991. doi: 10.1016/S1474-4422(19)30366-7. Lancet Neurol. 2019. PMID: 31609208 No abstract available.
The Biomarker Potential of miRNAs in Myotonic Dystrophy Type I.
Koehorst E, Ballester-Lopez A, Arechavala-Gomeza V, Martínez-Piñeiro A, Nogales-Gadea G. Koehorst E, et al. J Clin Med. 2020 Dec 4;9(12):3939. doi: 10.3390/jcm9123939. J Clin Med. 2020. PMID: 33291833 Free PMC article. Review.
Duchenne muscular dystrophy cell culture models created by CRISPR/Cas9 gene editing and their application in drug screening.
Soblechero-Martín P, Albiasu-Arteta E, Anton-Martinez A, de la Puente-Ovejero L, Garcia-Jimenez I, González-Iglesias G, Larrañaga-Aiestaran I, López-Martínez A, Poyatos-García J, Ruiz-Del-Yerro E, Gonzalez F, Arechavala-Gomeza V. Soblechero-Martín P, et al. Among authors: arechavala gomeza v. Sci Rep. 2021 Sep 14;11(1):18188. doi: 10.1038/s41598-021-97730-5. Sci Rep. 2021. PMID: 34521928 Free PMC article.
miR-106b is a novel target to promote muscle regeneration and restore satellite stem cell function in injured Duchenne dystrophic muscle.
Rodriguez-Outeiriño L, Hernandez-Torres F, Ramirez de Acuña F, Rastrojo A, Creus C, Carvajal A, Salmeron L, Montolio M, Soblechero-Martin P, Arechavala-Gomeza V, Franco D, Aranega AE. Rodriguez-Outeiriño L, et al. Among authors: arechavala gomeza v. Mol Ther Nucleic Acids. 2022 Aug 20;29:769-786. doi: 10.1016/j.omtn.2022.08.025. eCollection 2022 Sep 13. Mol Ther Nucleic Acids. 2022. PMID: 36159592 Free PMC article.
A multicenter comparison of quantification methods for antisense oligonucleotide-induced DMD exon 51 skipping in Duchenne muscular dystrophy cell cultures.
Hiller M, Falzarano MS, Garcia-Jimenez I, Sardone V, Verheul RC, Popplewell L, Anthony K, Ruiz-Del-Yerro E, Osman H, Goeman JJ, Mamchaoui K, Dickson G, Ferlini A, Muntoni F, Aartsma-Rus A, Arechavala-Gomeza V, Datson NA, Spitali P. Hiller M, et al. PLoS One. 2018 Oct 2;13(10):e0204485. doi: 10.1371/journal.pone.0204485. eCollection 2018. PLoS One. 2018. PMID: 30278058 Free PMC article.
41 results