Drug intelligence / Profile preview

antisense oligonucleotides targeting p53 cryptic exon

Development stage
Preclinical
Lead developer
Indiana University School of Medicine
Modality
Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
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Overview

Antisense oligonucleotides targeting the p53 cryptic exon are splice-switching oligonucleotides designed to modulate the expression of p53 isoforms. Developed by researchers at the Indiana University School of Medicine, these oligonucleotides target a stress-responsive cryptic exon embedded in the first intronic region of the p53 gene. Under stress conditions, a short transcript consisting of the first canonical exon and this cryptic exon is expressed, which normally limits canonical p53 expression. By targeting this cryptic exon or its transcription termination site, these antisense oligonucleotides can differentially control the expression of the short transcript and canonical p53, thereby enhancing canonical p53 expression to protect against ischemia-reperfusion injury and facilitate tissue recovery in acute kidney injury.

Other names
antisense oligonucleotides targeting p53 short isoformsplice-switching antisense oligonucleotides targeting p53 cryptic exon

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