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RNU4-2 antisense oligonucleotide
- Development stage
- Preclinical
- Lead developer
- n-Lorem Foundation
- 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
RNU4-2 antisense oligonucleotide is an investigational antisense oligonucleotide (ASO) therapy being developed for the treatment of ReNU syndrome (RNU4-2-related neurodevelopmental disorder). ReNU syndrome is a rare genetic neurodevelopmental condition caused by pathogenic variants in the non-coding RNU4-2 gene, which is transcribed into the U4 small nuclear RNA (snRNA) component of the major spliceosome. The ASO is designed to target the RNU4-2 (n.64_65insT) variant, which accounts for approximately 75% of ReNU syndrome cases, to correct the underlying genetic splicing defect and restore normal splicing.
- Other names
- RNU4-2 ASORNU-4-2 ASORNU 4-2 ASO
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