Mechanism of Splicing Regulation of Spinal Muscular …
The first approved drug for SMA, Nusinersen (Spinraza™), is an antisense oligonucleotide (ASO) that promotes inclusion of SMN2 exon 7 by sequestering an inhibitory cis -element called Intronic Splicing Silencer N1 or ISS-N1 [25, 26].
Discovery of a CNS penetrant small molecule SMN2 splicing …
Oct 15, 2020 · Through screening SMA patient-derived fibroblasts, a novel small molecule, designated TEC-1, was identified that selectively modulates SMN2 splicing over three …
- Author: Shiori Ando, Shunya Suzuki, Shoichi Okubo, Kazuki Ohuchi, Kei Takahashi, Shinsuke Nakamura, Masamits...
- Publish Year: 2020
The Antisense Oligonucleotide Nusinersen for Treatment of Spinal ...
Nusinersen is a survival motor neuron-2 (SMN2)-directed antisense oligonucleotide (ASO) designed to treat SMA caused by a deficiency in SMN proteins secondary from mutations in …
Antisense correction of SMN2 splicing in the CNS rescues necrosis …
Aug 1, 2010 · Previously, we identified a potent 2'-O- (2-methoxyethyl) (MOE) phosphorothioate-modified antisense oligonucleotide (ASO) that blocks an SMN2 intronic splicing silencer …
- Author: Yimin Hua, Kentaro Sahashi, Gene Hung, Frank Rigo, Marco A. Passini, C. Frank Bennett, Adrian R. Kra...
- Publish Year: 2010
Optimization of base editors for the functional correction of SMN2 …
Dec 6, 2023 · Targeting SMN2 transcripts with a small molecule or an antisense oligonucleotide (ASO) to transiently increase the retention of exon 7 showed notable clinical results in infants …
Antisense oligonucleotide therapy for neurodevelopmental disorders
The ASO Nusinersen is a general treatment for mutations of SMN1 in spinal muscular atrophy that corrects the splicing defect in the SMN2 gene. Milasen is a patient-specific ASO that …
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Antisense oligonucleotides and spinal muscular atrophy: skipping …
Aug 1, 2010 · Skordis et al. (2003) reported that an antisense oligonucleotide (ASO) that bound the same region of exon 7 and contained a tail capable of binding SR proteins enhanced …
The targeted nature of an ASO treatment SPINRAZA is designed to target a specific sequence in the survival motor neuron 2 (SMN2) pre-mRNA to modify splicing and increase functional SMN …
Antisense-Oligonucleotide Modulation of SMN2 Pre-mRNA Splicing
Jan 1, 2017 · Targeting SMN2 splicing to increase full-length protein expression is a promising approach for therapeutic intervention. Antisense oligonucleotide (ASO) technology offers a …
Counteracting chromatin effects of a splicing …
Jun 9, 2022 · Nusinersen is an antisense oligonucleotide (ASO) that upregulates E7 inclusion and SMN protein levels by displacing the splicing repressors hnRNPA1/A2 from their target site in intron 7. We show that by promoting …
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