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1.	Aoki, Y, et al. (author) Fine Tuning of Phosphorothioate Inclusion in 2'-O-Methyl Oligonucleotides Contributes to Specific Cell Targeting for Splice-Switching Modulation 2021 In: Frontiers in physiology. - : Frontiers Media SA. - 1664-042X. ; 12, s. 689179- Journal article (peer-reviewed)abstract Splice-switching antisense oligonucleotide- (SSO-) mediated correction of framedisrupting mutation-containing premessenger RNA (mRNA) transcripts using exon skipping is a highly promising treatment method for muscular diseases such as Duchenne muscular dystrophy (DMD). Phosphorothioate (PS) chemistry, a commonly used oligonucleotide modification, has been shown to increase the stability of and improve the pharmacokinetics of SSOs. However, the effect of PS inclusion in 2′-O-methyl SSOs (2OMe) on cellular uptake and splice switching is less well-understood. At present, we demonstrate that the modification of PS facilitates the uptake of 2OMe in H2k-mdx myoblasts. Furthermore, we found a dependency of SSO nuclear accumulation and high splice-switching activity on PS inclusion in 2OMe (2OMePS), as tested in various reporter cell lines carrying pLuc/705. Increased exon-inclusion activity was observed in muscle, neuronal, liver, and bone cell lineages via both the gymnotic uptake and lipofection of 2OMePS. Using the photoactivatable ribonucleoside-enhanced crosslinking and a subsequent proteomic approach, we identified several 2OMePS-binding proteins, which are likely to play a role in the trafficking of 2OMePS to the nucleus. Ablation of one of them, Ncl by small-interfering RNA (siRNA) enhanced 2OMePS uptake in C2C12 myoblasts and upregulated luciferase RNA splicing in the HeLa Luc/705 reporter cell line. Overall, we demonstrate that PS inclusion increases nuclear delivery and splice switching in muscle, neuronal, liver, and bone cell lineages and that the modulation of 2OMePS-binding partners may improve SSO delivery.
2.	Honcharenko, D, et al. (author) 2'-O-(N-(Aminoethyl)carbamoyl)methyl Modification Allows for Lower Phosphorothioate Content in Splice-Switching Oligonucleotides with Retained Activity 2022 In: Nucleic acid therapeutics. - 2159-3345. Journal article (peer-reviewed)
3.	Honcharenko, D, et al. (author) 2'-O-(N-(Aminoethyl)carbamoyl)methyl Modification Allows for Lower Phosphorothioate Content in Splice-Switching Oligonucleotides with Retained Activity 2022 In: Nucleic acid therapeutics. - : Mary Ann Liebert Inc. - 2159-3345 .- 2159-3337. ; 32:3, s. 221-233 Journal article (peer-reviewed)
4.	Milton, S, et al. (author) Nuclease resistant oligonucleotides with cell penetrating properties 2015 In: Chemical communications (Cambridge, England). - : Royal Society of Chemistry (RSC). - 1364-548X .- 1359-7345. ; 51:19, s. 4044-4047 Journal article (peer-reviewed)abstract 2′-O-AECM modified oligonucleotides provide a combination of remarkable properties, such as high stability towards degradation, stable duplexes with RNA and enhanced cellular uptake.
5.	Moreno, PMD, et al. (author) Development of bis-locked nucleic acid (bisLNA) oligonucleotides for efficient invasion of supercoiled duplex DNA 2013 In: Nucleic acids research. - : Oxford University Press (OUP). - 1362-4962 .- 0305-1048. ; 41:5, s. 3257-3273 Journal article (peer-reviewed)
6.	Rocha, CSJ, et al. (author) Four Novel Splice-Switch Reporter Cell Lines: Distinct Impact of Oligonucleotide Chemistry and Delivery Vector on Biological Activity 2016 In: Nucleic acid therapeutics. - : Mary Ann Liebert Inc. - 2159-3345 .- 2159-3337. ; 26:6, s. 381-391 Journal article (peer-reviewed)
7.	Rocha, CSJ, et al. (author) RNA therapeutics inactivate PCSK9 by inducing a unique intracellular retention form 2015 In: Journal of molecular and cellular cardiology. - : Elsevier BV. - 1095-8584 .- 0022-2828. ; 82, s. 186-193 Journal article (peer-reviewed)
8.	Saher, O, et al. (author) Novel peptide-dendrimer/lipid/oligonucleotide ternary complexes for efficient cellular uptake and improved splice-switching activity 2018 In: European journal of pharmaceutics and biopharmaceutics : official journal of Arbeitsgemeinschaft fur Pharmazeutische Verfahrenstechnik e.V. - : Elsevier BV. - 1873-3441. ; 132, s. 29-40 Journal article (peer-reviewed)

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Result 1-8 of 8

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Type of publication: journal article (8)

Type of content: peer-reviewed (8)

Author/Editor: Rocha, CSJ (8); Smith, CIE (7); Lundin, KE (6); Zain, R (5); EL-Andaloussi, S (4); Stromberg, R (3); show more...; Moreno, PMD (3); Milton, S (3); Honcharenko, D. (3); Behlke, MA (2); Wiklander, OPB (2); Tedebark, U (2); Slaitas, A (2); Murtola, M (2); Honcharenko, M (2); Maity, J (2); Aoki, M. (1); Wengel, J. (1); Larsson, L (1); Johansson, H (1); Parini, P (1); Graham, M. (1); Zaghloul, EM (1); Gupta, D. (1); Lehto, T (1); Nordin, JZ (1); Aoki, Y. (1); Sathyaprakash, C (1); Mager, I (1); Ezzat, K (1); Wood, MJA (1); Miyatake, S (1); Hashimoto, Y (1); Roberts, TC (1); Strömberg, R. (1); Bestas, B (1); Saher, O (1); Bergquist, H (1); Hollfelder, F. (1); Oprea, II (1); Zamolo, S (1); Heitz, M (1); Darbre, T (1); Reymond, JL (1); Pabon, YV (1); Geny, S (1); Jorgensen, PT (1); Pedersen, EB (1); Zaghioul, EM (1); show less...

University: Karolinska Institutet (8)

Language: English (8)

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