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Page 1
An oral antisense oligonucleotide for PCSK9 inhibition.
Gennemark P, Walter K, Clemmensen N, Rekić D, Nilsson CAM, Knöchel J, Hölttä M, Wernevik L, Rosengren B, Kakol-Palm D, Wang Y, Yu RZ, Geary RS, Riney SJ, Monia BP, Isaksson R, Jansson-Löfmark R, Rocha CSJ, Lindén D, Hurt-Camejo E, Crooke R, Tillman L, Rydén-Bergsten T, Carlsson B, Andersson U, Elebring M, Tivesten A, Davies N. Gennemark P, et al. Among authors: rocha csj. Sci Transl Med. 2021 May 12;13(593):eabe9117. doi: 10.1126/scitranslmed.abe9117. Sci Transl Med. 2021. PMID: 33980578
Novel peptide-dendrimer/lipid/oligonucleotide ternary complexes for efficient cellular uptake and improved splice-switching activity.
Saher O, Rocha CSJ, Zaghloul EM, Wiklander OPB, Zamolo S, Heitz M, Ezzat K, Gupta D, Reymond JL, Zain R, Hollfelder F, Darbre T, Lundin KE, El Andaloussi S, Smith CIE. Saher O, et al. Among authors: rocha csj. Eur J Pharm Biopharm. 2018 Nov;132:29-40. doi: 10.1016/j.ejpb.2018.09.002. Epub 2018 Sep 4. Eur J Pharm Biopharm. 2018. PMID: 30193928
2'-O-(N-(Aminoethyl)carbamoyl)methyl Modification Allows for Lower Phosphorothioate Content in Splice-Switching Oligonucleotides with Retained Activity.
Honcharenko D, Rocha CSJ, Lundin KE, Maity J, Milton S, Tedebark U, Murtola M, Honcharenko M, Slaitas A, Smith CIE, Zain R, Strömberg R. Honcharenko D, et al. Among authors: rocha csj. Nucleic Acid Ther. 2022 Jun;32(3):221-233. doi: 10.1089/nat.2021.0086. Epub 2022 Mar 2. Nucleic Acid Ther. 2022. PMID: 35238623 Free PMC article.
Fine Tuning of Phosphorothioate Inclusion in 2'-O-Methyl Oligonucleotides Contributes to Specific Cell Targeting for Splice-Switching Modulation.
Aoki Y, Rocha CSJ, Lehto T, Miyatake S, Johansson H, Hashimoto Y, Nordin JZ, Mager I, Aoki M, Graham M, Sathyaprakash C, Roberts TC, Wood MJA, Behlke MA, Andaloussi SE. Aoki Y, et al. Among authors: rocha csj. Front Physiol. 2021 Oct 13;12:689179. doi: 10.3389/fphys.2021.689179. eCollection 2021. Front Physiol. 2021. PMID: 34721051 Free PMC article.