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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: buijsen ram. 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.
Human Induced Pluripotent Stem Cell-Based Modelling of Spinocerebellar Ataxias.
Hommersom MP, Buijsen RAM, van Roon-Mom WMC, van de Warrenburg BPC, van Bokhoven H. Hommersom MP, et al. Among authors: buijsen ram. Stem Cell Rev Rep. 2022 Feb;18(2):441-456. doi: 10.1007/s12015-021-10184-0. Epub 2021 May 25. Stem Cell Rev Rep. 2022. PMID: 34031815 Free PMC article. Review.
Antisense Oligonucleotide-Induced Amyloid Precursor Protein Splicing Modulation as a Therapeutic Approach for Dutch-Type Cerebral Amyloid Angiopathy.
Daoutsali E, Hailu TT, Buijsen RAM, Pepers BA, van der Graaf LM, Verbeek MM, Curtis D, de Vlaam T, van Roon-Mom WMC. Daoutsali E, et al. Among authors: buijsen ram. Nucleic Acid Ther. 2021 Oct;31(5):351-363. doi: 10.1089/nat.2021.0005. Epub 2021 May 31. Nucleic Acid Ther. 2021. PMID: 34061681 Free PMC article.
Therapeutic Strategies for Spinocerebellar Ataxia Type 1.
Kerkhof LMC, van de Warrenburg BPC, van Roon-Mom WMC, Buijsen RAM. Kerkhof LMC, et al. Among authors: buijsen ram. Biomolecules. 2023 May 2;13(5):788. doi: 10.3390/biom13050788. Biomolecules. 2023. PMID: 37238658 Free PMC article. Review.
Antisense oligonucleotide-mediated disruption of HTT caspase-6 cleavage site ameliorates the phenotype of YAC128 Huntington disease mice.
Kuijper EC, Overzier M, Suidgeest E, Dzyubachyk O, Maguin C, Pérot JB, Flament J, Ariyurek Y, Mei H, Buijsen RAM, van der Weerd L, van Roon-Mom W. Kuijper EC, et al. Among authors: buijsen ram. Neurobiol Dis. 2024 Jan;190:106368. doi: 10.1016/j.nbd.2023.106368. Epub 2023 Nov 29. Neurobiol Dis. 2024. PMID: 38040383 Free article.
Generation of 3 spinocerebellar ataxia type 1 (SCA1) patient-derived induced pluripotent stem cell lines LUMCi002-A, B, and C and 2 unaffected sibling control induced pluripotent stem cell lines LUMCi003-A and B.
Buijsen RAM, Gardiner SL, Bouma MJ, van der Graaf LM, Boogaard MW, Pepers BA, Eussen B, de Klein A, Freund C, van Roon-Mom WMC. Buijsen RAM, et al. Stem Cell Res. 2018 May;29:125-128. doi: 10.1016/j.scr.2018.03.018. Epub 2018 Apr 5. Stem Cell Res. 2018. PMID: 29656178 Free article.
Generation of 3 human induced pluripotent stem cell lines LUMCi005-A, B and C from a Hereditary Cerebral Hemorrhage with Amyloidosis-Dutch type patient.
Daoutsali E, Buijsen RAM, van de Pas S, Jong A', Mikkers H, Brands T, Eussen B, de Klein A, van der Graaf LM, Pepers BA, Freund C, Terwindt GM, Orlova VV, van Roon-Mom WMC. Daoutsali E, et al. Among authors: buijsen ram. Stem Cell Res. 2019 Jan;34:101359. doi: 10.1016/j.scr.2018.101359. Epub 2018 Dec 14. Stem Cell Res. 2019. PMID: 30611017 Free article. English.
32 results