334 related articles for article (PubMed ID: 34445083)
1. High Concentration of an ISS-N1-Targeting Antisense Oligonucleotide Causes Massive Perturbation of the Transcriptome.
Ottesen EW; Luo D; Singh NN; Singh RN
Int J Mol Sci; 2021 Aug; 22(16):. PubMed ID: 34445083
[TBL] [Abstract][Full Text] [Related]
2. 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]
3. Splicing of a critical exon of human Survival Motor Neuron is regulated by a unique silencer element located in the last intron.
Singh NK; Singh NN; Androphy EJ; Singh RN
Mol Cell Biol; 2006 Feb; 26(4):1333-46. PubMed ID: 16449646
[TBL] [Abstract][Full Text] [Related]
4. How the discovery of ISS-N1 led to the first medical therapy for spinal muscular atrophy.
Singh NN; Howell MD; Androphy EJ; Singh RN
Gene Ther; 2017 Sep; 24(9):520-526. PubMed ID: 28485722
[TBL] [Abstract][Full Text] [Related]
5. 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]
6. Splicing regulation in spinal muscular atrophy by an RNA structure formed by long-distance interactions.
Singh NN; Lee BM; Singh RN
Ann N Y Acad Sci; 2015 Apr; 1341():176-87. PubMed ID: 25727246
[TBL] [Abstract][Full Text] [Related]
7. 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]
8. Bifunctional RNAs targeting the intronic splicing silencer N1 increase SMN levels and reduce disease severity in an animal model of spinal muscular atrophy.
Osman EY; Yen PF; Lorson CL
Mol Ther; 2012 Jan; 20(1):119-26. PubMed ID: 22031236
[TBL] [Abstract][Full Text] [Related]
9. Recent Advances and Clinical Applications of Exon Inclusion for Spinal Muscular Atrophy.
Son HW; Yokota T
Methods Mol Biol; 2018; 1828():57-68. PubMed ID: 30171534
[TBL] [Abstract][Full Text] [Related]
10. High Concentration or Combined Treatment of Antisense Oligonucleotides for Spinal Muscular Atrophy Perturbed
Wijaya YOS; Niba ETE; Nishio H; Okamoto K; Awano H; Saito T; Takeshima Y; Shinohara M
Genes (Basel); 2022 Apr; 13(4):. PubMed ID: 35456491
[TBL] [Abstract][Full Text] [Related]
11. Antisense oligonucleotide mediated therapy of spinal muscular atrophy.
Sivanesan S; Howell MD; Didonato CJ; Singh RN
Transl Neurosci; 2013 Mar; 4(1):. PubMed ID: 24265944
[TBL] [Abstract][Full Text] [Related]
12. RNA in spinal muscular atrophy: therapeutic implications of targeting.
Singh RN; Seo J; Singh NN
Expert Opin Ther Targets; 2020 Aug; 24(8):731-743. PubMed ID: 32538213
[TBL] [Abstract][Full Text] [Related]
13. Effect of combined systemic and local morpholino treatment on the spinal muscular atrophy Δ7 mouse model phenotype.
Nizzardo M; Simone C; Salani S; Ruepp MD; Rizzo F; Ruggieri M; Zanetta C; Brajkovic S; Moulton HM; Müehlemann O; Bresolin N; Comi GP; Corti S
Clin Ther; 2014 Mar; 36(3):340-56.e5. PubMed ID: 24636820
[TBL] [Abstract][Full Text] [Related]
14. Antisense masking of an hnRNP A1/A2 intronic splicing silencer corrects SMN2 splicing in transgenic mice.
Hua Y; Vickers TA; Okunola HL; Bennett CF; Krainer AR
Am J Hum Genet; 2008 Apr; 82(4):834-48. PubMed ID: 18371932
[TBL] [Abstract][Full Text] [Related]
15. Pre-mRNA Splicing Modulation by Antisense Oligonucleotides.
Singh NN; Luo D; Singh RN
Methods Mol Biol; 2018; 1828():415-437. PubMed ID: 30171557
[TBL] [Abstract][Full Text] [Related]
16. 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]
17. Mechanism of Splicing Regulation of Spinal Muscular Atrophy Genes.
Singh RN; Singh NN
Adv Neurobiol; 2018; 20():31-61. PubMed ID: 29916015
[TBL] [Abstract][Full Text] [Related]
18. Nusinersen: antisense oligonucleotide to increase SMN protein production in spinal muscular atrophy.
Paton DM
Drugs Today (Barc); 2017 Jun; 53(6):327-337. PubMed ID: 28799578
[TBL] [Abstract][Full Text] [Related]
19. Evolving concepts on human SMN pre-mRNA splicing.
Singh RN
RNA Biol; 2007; 4(1):7-10. PubMed ID: 17592254
[TBL] [Abstract][Full Text] [Related]
20. ISS-N1 makes the First FDA-approved Drug for Spinal Muscular Atrophy.
Ottesen EW
Transl Neurosci; 2017 Jan; 8():1-6. PubMed ID: 28400976
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]