These tools will no longer be maintained as of December 31, 2024. Archived website can be found here. PubMed4Hh GitHub repository can be found here. Contact NLM Customer Service if you have questions.
9. NCALD Antisense Oligonucleotide Therapy in Addition to Nusinersen further Ameliorates Spinal Muscular Atrophy in Mice. Torres-Benito L; Schneider S; Rombo R; Ling KK; Grysko V; Upadhyay A; Kononenko NL; Rigo F; Bennett CF; Wirth B Am J Hum Genet; 2019 Jul; 105(1):221-230. PubMed ID: 31230718 [TBL] [Abstract][Full Text] [Related]
10. The Antisense Transcript SMN-AS1 Regulates SMN Expression and Is a Novel Therapeutic Target for Spinal Muscular Atrophy. d'Ydewalle C; Ramos DM; Pyles NJ; Ng SY; Gorz M; Pilato CM; Ling K; Kong L; Ward AJ; Rubin LL; Rigo F; Bennett CF; Sumner CJ Neuron; 2017 Jan; 93(1):66-79. PubMed ID: 28017471 [TBL] [Abstract][Full Text] [Related]
11. Combined treatment with the histone deacetylase inhibitor LBH589 and a splice-switch antisense oligonucleotide enhances SMN2 splicing and SMN expression in Spinal Muscular Atrophy cells. Pagliarini V; Guerra M; Di Rosa V; Compagnucci C; Sette C J Neurochem; 2020 Apr; 153(2):264-275. PubMed ID: 31811660 [TBL] [Abstract][Full Text] [Related]
12. Challenges and future perspective of antisense therapy for spinal muscular atrophy: A review. Nakevska Z; Yokota T Eur J Cell Biol; 2023 Jun; 102(2):151326. PubMed ID: 37295266 [TBL] [Abstract][Full Text] [Related]
13. Enhancing Antisense Oligonucleotide-Based Therapeutic Delivery with DG9, a Versatile Cell-Penetrating Peptide. Haque US; Yokota T Cells; 2023 Oct; 12(19):. PubMed ID: 37830609 [TBL] [Abstract][Full Text] [Related]
14. Combination of valproic acid and morpholino splice-switching oligonucleotide produces improved outcomes in spinal muscular atrophy patient-derived fibroblasts. Farrelly-Rosch A; Lau CL; Patil N; Turner BJ; Shabanpoor F Neurochem Int; 2017 Sep; 108():213-221. PubMed ID: 28389270 [TBL] [Abstract][Full Text] [Related]
15. A short antisense oligonucleotide masking a unique intronic motif prevents skipping of a critical exon in spinal muscular atrophy. Singh NN; Shishimorova M; Cao LC; Gangwani L; Singh RN RNA Biol; 2009; 6(3):341-50. PubMed ID: 19430205 [TBL] [Abstract][Full Text] [Related]
16. Mechanistic principles of antisense targets for the treatment of spinal muscular atrophy. Singh NN; Lee BM; DiDonato CJ; Singh RN Future Med Chem; 2015; 7(13):1793-808. PubMed ID: 26381381 [TBL] [Abstract][Full Text] [Related]
18. Evaluating Efficacy of Peptide-Delivered Oligonucleotides Using the Severe Taiwanese SMA Mouse Model. Goli L; Stoodley J; Hammond SM; Raz R Methods Mol Biol; 2022; 2383():491-513. PubMed ID: 34766309 [TBL] [Abstract][Full Text] [Related]
19. Spinal muscular atrophy: antisense oligonucleotide therapy opens the door to an integrated therapeutic landscape. Wood MJA; Talbot K; Bowerman M Hum Mol Genet; 2017 Oct; 26(R2):R151-R159. PubMed ID: 28977438 [TBL] [Abstract][Full Text] [Related]
20. Nusinersen in the Treatment of Spinal Muscular Atrophy. Goodkey K; Aslesh T; Maruyama R; Yokota T Methods Mol Biol; 2018; 1828():69-76. PubMed ID: 30171535 [TBL] [Abstract][Full Text] [Related] [Next] [New Search]