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UBE3A ASO
- Development stage
- Preclinical
- Lead developer
- Quiver Bioscience
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intracerebroventricular, Intrathecal, Intravenous, In Utero Intracranial, Intra-amniotic
01
Overview
Quiver Bioscience is developing an antisense oligonucleotide (ASO) therapeutic targeting UBE3A for the treatment of Dup15q syndrome, a rare neurodevelopmental disorder. Dup15q syndrome is characterized by the duplication of the 15q11.2-q13.1 chromosomal region, leading to the overexpression of the UBE3A protein, which results in symptoms such as epilepsy, autism spectrum disorder, and intellectual disability. The ASO is designed to reduce UBE3A expression levels to rescue patient-specific cellular phenotypes. The program utilizes Quiver's Genomic Positioning System (GPS) platform, which integrates human single-cell neuronal electrophysiology with artificial intelligence and machine learning to identify and optimize ASO candidates.
- Other names
- UBE3A antisense oligonucleotideUBE-3A antisense oligonucleotideUBE 3A antisense oligonucleotideUBE3A ASOUBE-3A ASOUBE 3A ASO
02
Targets
UBE3A-ATS (UBE3A antisense transcript)
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