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Delivery of oligonucleotide-based therapeutics: challenges and opportunities.
Hammond SM, Aartsma-Rus A, Alves S, Borgos SE, Buijsen RAM, Collin RWJ, Covello G, Denti MA, Desviat LR, Echevarría L, Foged C, Gaina G, Garanto A, Goyenvalle AT, Guzowska M, Holodnuka I, Jones DR, Krause S, Lehto T, Montolio M, Van Roon-Mom W, Arechavala-Gomeza V. Hammond SM, et al. Among authors: desviat lr. EMBO Mol Med. 2021 Apr 9;13(4):e13243. doi: 10.15252/emmm.202013243. Epub 2021 Apr 6. EMBO Mol Med. 2021. PMID: 33821570 Free PMC article. Review.
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. Among authors: desviat lr. 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.
Generation of a gene-corrected human isogenic line (UAMi006-A) from propionic acidemia patient iPSC with an homozygous mutation in the PCCB gene using CRISPR/Cas9 technology.
Fulgencio-Covián A, Álvarez M, Pepers BA, López-Márquez A, Ugarte M, Pérez B, van Roon-Mom WMC, Desviat LR, Richard E. Fulgencio-Covián A, et al. Among authors: desviat lr. Stem Cell Res. 2020 Dec;49:102055. doi: 10.1016/j.scr.2020.102055. Epub 2020 Oct 17. Stem Cell Res. 2020. PMID: 33128956 Free article.
RNA solutions to treat inborn errors of metabolism.
Martinez-Pizarro A, Desviat LR. Martinez-Pizarro A, et al. Among authors: desviat lr. Mol Genet Metab. 2022 Aug;136(4):289-295. doi: 10.1016/j.ymgme.2022.07.006. Epub 2022 Jul 8. Mol Genet Metab. 2022. PMID: 35849888 Free PMC article. Review.
156 results