Metabolomics,Unknown,Transcriptomics,Genomics,Proteomics

Dataset Information

0

Antisense oligonucleotides targeting nuclear RNA for in vivo correction of myotonic dystrophy


ABSTRACT: This study examined the effects of antisense oligonucleotides (ASOs) on the muscle transcriptome in a transgenic mouse model of myotonic dystrophy. Two ASOs were tested in HSALR transgenic mice. Both of the ASOs targeted mRNA from a human skeletal actin (hACTA1) transgene. This transgene contains an expanded CTG repeat in the 3M-bM-^@M-^Y untranslated region (UTR) (Mankodi et al M-bM-^@M-^\Myotonic dystrophy in transgenic mice expressing an expanded CUG repeatM-bM-^@M-^] Science 2000; 289:1769-72). ASO 445235 targeted the hACTA1 transcript in the 3M-bM-^@M-^Y UTR, downstream from the expanded repeat. ASO 190401 targeted the hACTA1 transcript in the coding region. The HSALR mice received 4 weeks of biweekly subcutaneous injections of vehicle (saline), ASO 190401, or ASO 445236 (n = 4 per group), at a dose of 25 mg/kg per injection. Wild-type mice of the same strain background received saline injections (n = 4). One week after the final injection, quadriceps muscle was harvested for RNA analysis. Four conditions, four arrays per condition, to compare WT and HSALR trangenic mice without treatment (saline) and to examine the effect of two oligos (vs. saline) in the HSALR transgenic mice.

ORGANISM(S): Mus musculus

SUBMITTER: Stephen Welle 

PROVIDER: E-GEOD-38962 | biostudies-arrayexpress |

REPOSITORIES: biostudies-arrayexpress

Similar Datasets

2012-06-28 | GSE38962 | GEO
2023-05-15 | GSE220777 | GEO
2021-12-08 | GSE190473 | GEO
2022-04-26 | GSE201237 | GEO
2016-09-08 | E-GEOD-77936 | biostudies-arrayexpress
2024-01-08 | E-MTAB-11589 | biostudies-arrayexpress
2016-09-08 | E-GEOD-77935 | biostudies-arrayexpress
2022-07-12 | GSE197166 | GEO
2021-12-15 | GSE188684 | GEO
2022-10-15 | PXD035704 | Pride