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antisense oligonucleotide targeting endoglin (ENG) splicing
- Development stage
- Preclinical
- Lead developer
- Leiden University Medical Center
- 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
01
Overview
Antisense oligonucleotides (ASOs) targeting endoglin (ENG) splicing are designed to modulate the alternative splicing of the ENG pre-mRNA. Specifically, these ASOs promote terminal intron retention to shift the isoform ratio from the pro-fibrotic long endoglin (l-ENG) isoform toward the anti-fibrotic short endoglin (s-ENG) isoform. Developed by researchers at the Leiden University Medical Center, this therapeutic approach aims to reduce transforming growth factor-beta 1 (TGF-β1) downstream signaling and extracellular matrix production, offering a potential strategy to treat renal interstitial fibrosis and slow the progression of chronic kidney disease (CKD) and chronic allograft dysfunction (CAD).
- Other names
- ENG splicing-targeting ASOENG splicing-targeting antisense oligonucleotideendoglin splicing-targeting ASOendoglin splicing-targeting antisense oligonucleotide
02
Targets
ENG (Integrin Beta 6)
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