Antisense correction of SMN2 exon 7 splicing

NIAID Data Ecosystem2026-03-12 收录

基因剪接调控

反义寡核苷酸

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In this study, we seek to characterize the on- and off-target effects of an antisense oligonucleotide targeting the ISS-N1 element for correction of SMN2 exon 7 splicing

创建时间：

2021-08-24

Antisense correction of SMN2 exon 7 splicing

基因剪接调控

反义寡核苷酸

In this study, we seek to characterize the on- and off-target effects of an antisense oligonucleotide targeting the ISS-N1 element for correction of SMN2 exon 7 splicing

NIAID Data Ecosystem10

Statistical prediction of single-stranded regions in RNA secondary structure and application to predicting effective antisense target sites and beyond

RNA结构预测

反义寡核苷酸

Single-stranded regions in RNA secondary structure are important for RNA–RNA and RNA–protein interactions. We present a probability profile approach for the prediction of these regions based on a stat

PubMed Central2001-03-01 更新10

Distance to the Nearest Exon of Intronic SNPs Tends to Be Smaller Than the Distance to the Skipped Exon of ISE SNPs.

基因组变异分析

基因剪接调控

The upper 3 rows show the general distribution of the distance to the nearest exon of all intronic SNPs and the distance to the skipped exon of the ISE SNPs (Figure S3). The bottom 3 rows show the dis

Figshare2015-12-02 更新10

HuR antagonizes the effect of an intronic pyrimidine-rich sequence in regulating WT1 +/−KTS isoforms

基因剪接调控

RNA-蛋白质相互作用

ABSTRACT WT1 +KTS and -KTS isoforms only differ in three amino acids in protein sequence but show significant functional difference. The +/−KTS isoforms were generated by alternative usage of two adja

NIAID Data Ecosystem10

Reversal of MECP2 duplication syndrome using genetic rescue and antisense oligonucleotides [ASO time point 2]. Mus musculus

遗传疾病治疗

反义寡核苷酸

MECP2 duplication syndrome, a childhood neurological disorder characterized by autism, intellectual disability, motor dysfunction, anxiety and epilepsy, is caused by a duplication on chromosome Xq28 s

NIAID Data Ecosystem10

Antisense correction of SMN2 exon 7 splicing

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