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Head to head comparison of target knockdown following OTV vs. anti-TfR:ASO administration in mice

ABSTRACT: Head to head comparison of target knockdown following OTV vs. anti-TfR:ASO administration in mice

PROVIDER: PRJNA833038 | ENA |

REPOSITORIES: ENA

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			Action	DRS
	SRR18963062.fastq.gz	Fastqsanger.gz
	SRR18963063.fastq.gz	Fastqsanger.gz
	SRR18963064.fastq.gz	Fastqsanger.gz
	SRR18963065.fastq.gz	Fastqsanger.gz
	SRR18963066.fastq.gz	Fastqsanger.gz

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Head to head comparison of target knockdown following OTV vs. anti-TfR:ASO administration in mice

Project description:Using single nucleus RNA sequencing, we determined that peripheral administration of OTV:Malat1 results in much more widespread and robust target knockdown throughout the mouse brain compared to anti-TfR:Malat1.

2024-02-12 | GSE201787 | GEO

A Biodistribution Study of OTV vs. ASO by Intravenous or Intrathecal Administration in Cynomolgus Monkeys

Project description:The objective of this study is to evaluate ASO biodistribution and pharmacodynamic response following intravenous delivery of conjugated OTV, which utilizes transferrin-receptor binding to deliver ASO, vs. intrathecal delivery of naked ASO in cynomolgus monkeys, the current gold standard for ASO delivery. Three groups were included for comparison: 1) Negative control cohort = Four biweekly intravenous doses of saline (n=3); 2) IT cohort = Single intrathecal dose of 4mg MALAT1 ASO (n=3); 3)  OTV multi dose cohort = Four biweekly intravenous doses of 30mpk OTV:MALAT1 (n=3). Tissues were collected two weeks after the final dose to allow sufficient time for target knockdown. Brain, spinal cord, and peripheral tissues were dissected and frozen for molecular analysis. Compared to an intrathecal injection of ASO, systemic administration of OTV resulted in significantly more homogenous biodistribution of ASO in the central nervous system as measured by a probe-based hybridization assay as well as staining with an anti-ASO antibody. One potential consequence of heterogeneous ASO distribution is heterogeneous target knockdown throughout the CNS. We observed more consistent target knockdown throughout the cortex, subcortical areas, and spinal cord of monkeys dosed peripherally with OTV. By contrast, monkeys dosed intrathecally with naked ASO showed much more knockdown in the spinal cord compared to the brian, consistent with ASO distribution.

2024-02-12 | GSE208114 | GEO

A Biodistribution Study of OTV vs. ASO by Intravenous or Intrathecal Administration in Cynomolgus Monkeys

Project description:A Biodistribution Study of OTV vs. ASO by Intravenous or Intrathecal Administration in Cynomolgus Monkeys

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Gene expression profiling of HCCLM3 cells: Control ASO vs SCARNA13 ASO

Project description:Gene expression profiling of HCCLM3 cells: Control ASO vs SCARNA13 ASO

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HF liver: Control vs. Plin2-ASO treated

Project description:Transcriptional profiling of mouse liver comparing control, scrambled-oligonucleotides (Cont)-treated mice with Perilipin 2-antisense oligonucleotides (Plin2-ASO)-treated mice. C57BL/6 mice on a high-fat diet were treated with oligonucleotides in vivo. The goal was to determine the effects of Plin2 down-regulation in the liver on global gene expression. Two-condition experiment, control oligonucleotides vs. Plin2-antisense oligonucleotides. Biological replicates: 4 control replicates, 4 Plin2-ASO replicates.

2012-12-05 | E-GEOD-37011 | biostudies-arrayexpress

HF liver: Control vs. Plin2-ASO treated

2012-12-05 | GSE37011 | GEO

ASO-mediated RNA Suppression through Targeted Epigenetic Reprogramming of the Hdac2 Gene

Project description:We elicited ASO-mediated knockdown of mRNAs in the nervous system by targeting the memory-repressor gene Histone deacetylase 2 (Hdac2) in vitro and in vivo. We observed that an Hdac2-targeted ASO not only triggered a reduction in mRNA levels, but also a direct transcriptional suppression of the Hdac2 gene in neurons in vitro. In further in vivo studies, we observed that a single dose of the Hdac2-targeted ASO delivered by intracerebroventricular injection into the central nervous system (CNS) achieved steady-state diminution of Hdac2 mRNA levels that lasted more than 6 months in vivo. Knockdown of this factor resulted in memory enhancement. ASO administration also caused a prolonged alteration of steady-state levels of a set of known memory-associated mRNAs and triggered secondary changes in the epigenome at sites outside of the primary target gene.

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Project description:InVitro Ex-Tfr RNAseq

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Decreased TFR/TFH ratio in SIV-infected rhesus macaques may contribute to accumulation of TFH cells in chronic infection

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2015-09-02 | E-GEOD-69756 | biostudies-arrayexpress

Regulation of TFR cells by Blimp1

Project description:What genes does the transcription factor Blimp1 regulate in Tfr cells?

2020-01-07 | GSE143146 | GEO