MBNL splicing activity depends on RNA binding site structural context
Taylor K, Sznajder LJ, Cywoniuk P, Thomas JD, Swanson MS, Sobczak K. Nucleic Acids Res. 2018 Jun 28. 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
Antisense oligonucleotides correct the familial dysautonomia splicing defect in IKBKAP transgenic mice
Sinha R, Kim YJ, Nomakuchi T, Sahashi K, Hua Y, Rigo F, Bennett CF, Krainer AR. Nucleic Acids Res. 2018 Apr 17. PubMed
Machine Learning To Predict Cell-Penetrating Peptides for Antisense Delivery
Wolfe JM, Fadzen CM, Choo ZN, Holden RL, Yao M, Hanson GJ, Pentelute BL. ACS Cent Sci. 2018 Apr 25;4(4):512-520. PubMed
Systemic administration of the antisense oligonucleotide NS-065/NCNP-01 for skipping of exon 53 in patients with Duchenne muscular dystrophy
Komaki H, Nagata T, Saito T, Masuda S, Takeshita E, Sasaki M, Tachimori H, Nakamura H, Aoki Y, Takeda S. Sci Transl Med. 2018 Apr 18;10(437). PubMed
Systematic Analysis of Splice-Site-Creating Mutations in Cancer
Jayasinghe RG, Cao S, Gao Q, Wendl MC, Vo NS, Reynolds SM, Zhao Y, Climente-GonzĂ¡lez H, Chai S, Wang F, Varghese R, Huang M, Liang WW, Wyczalkowski MA, Sengupta S, Li Z, Payne SH, Fenyö D, Miner JH, Walter MJ; Cancer Genome Atlas Research Network, ...
Measuring DNA hybridization using fluorescent DNA-stabilized silver clusters to investigate mismatch effects on therapeutic oligonucleotides
de Bruin D, Bossert N, Aartsma-Rus A, Bouwmeester D. J Nanobiotechnology. 2018 Apr 6;16(1):37. PubMed
Targeting Amyloid-β Precursor Protein, APP, Splicing with Antisense Oligonucleotides Reduces Toxic Amyloid-β Production
Chang JL, Hinrich AJ, Roman B, Norrbom M, Rigo F, Marr RA, Norstrom EM, Hastings ML. MolTher. 2018 Mar 6. pii: S1525-0016(18)30105-9. PubMed
RNA-Seq Analysis of an Antisense Sequence Optimized for Exon Skipping in Duchenne Patients Reveals No Off-Target Effect
Domenger C, Allais M, François V, Léger A, Lecomte E, Montus M, Servais L, Voit T, Moullier P, Audic Y, Le Guiner C. MolTher Nucleic Acids. 2018 Mar 2;10:277-291. PubMed