NS-065/NCNP-01: An Antisense Oligonucleotide for Potential Treatment of Exon 53 Skipping in Duchenne Muscular Dystrophy
Watanabe N, Nagata T, Satou Y, Masuda S, Saito T, Kitagawa H, Komaki H, Takagaki K, Takeda S. Mol Ther Nucleic Acids. 2018 Dec 7;13:442-449. PubMed
Modulation of MKNK2 alternative splicing by splice-switching oligonucleotides as a novel approach for glioblastoma treatment
Mogilevsky M, Shimshon O, Kumar S, Mogilevsky A, Keshet E, Yavin E, Heyd F, Karni R. Nucleic Acids Res. 2018 Oct 17. PubMed
Splice-Modulating Oligonucleotide QR-110 Restores CEP290 mRNA and Function in Human c.2991+1655A>G LCA10 Models
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Morpholino Antisense Oligomers as a Potential Therapeutic Option for the Correction of Alternative Splicing in PMD, SPG2, and HEMS
Tantzer S, Sperle K, Kenaley K, Taube J, Hobson GM. Mol Ther Nucleic Acids. 2018 Sep 7;12:420-432. PubMed
A platform for discovery of functional cell-penetrating peptides for efficient multi-cargo intracellular delivery
Hoffmann K, Milech N, Juraja SM, Cunningham PT, Stone SR, Francis RW, Anastasas M, Hall CM, Heinrich T, Bogdawa HM, Winslow S, Scobie MN, Dewhurst RE, Florez L, Ong F, Kerfoot M, Champain D, Adams AM, Fletcher S, Viola HM, Hool LC, Connor T, Longville ...
A CCDC50 splice variant is modulated by SRSF3 and promotes hepatocellular carcinoma via the Ras signaling pathway
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RNA Structure Design Improves Activity and Specificity of trans-Splicing-Triggered Cell Death in a Suicide Gene Therapy Approach
Poddar S, Loh PS, Ooi ZH, Osman F, Eul J, Patzel V. Mol Ther Nucleic Acids. 2018 Jun 1;11:41-56. PubMed
Peptide-conjugated phosphodiamidate oligomer-mediated exon skipping has benefits for cardiac function in mdx and Cmah-/-mdx mouse models of Duchenne muscular dystrophy
Blain AM, Greally E, McClorey G, Manzano R, Betts CA, Godfrey C, O'Donovan L, Coursindel T, Gait MJ, Wood MJ, MacGowan GA, Straub VW. PLoS One. 2018 Jun 18;13(6):e0198897. PubMed
Efficient exon skipping of SGCG mutations mediated by phosphorodiamidate morpholino oligomers
Wyatt EJ, Demonbreun AR, Kim EY, Puckelwartz MJ, Vo AH, Dellefave-Castillo LM, Gao QQ, Vainzof M, Pavanello RCM, Zatz M, McNally EM. JCI Insight. 2018 May 3;3(9). pii: 99357. PubMed
Blocking of an intronic splicing silencer completely rescues IKBKAP exon 20 splicing in familial dysautonomia patient cells
Bruun GH, Bang JM, Christensen LL, Brøner S, Petersen US, Guerra B, Grønning AG, Doktor TK, Andresen BS. Nucleic Acids Res. 2018 May 14. PubMed