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C1q antisense oligonucleotide
- Development stage
- Preclinical
- Lead developer
- UMass Chan Medical School
- 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
C1q antisense oligonucleotide (also referred to as C1qa ASO) is an antisense oligonucleotide (ASO) designed to knockdown the expression of the C1qa gene, which encodes the A chain of complement component 1q (C1q). Developed by researchers at the University of Massachusetts Chan Medical School in a collaboration between the labs of Dorothy P. Schafer and Jonathan K. Watts, this RNA-based therapeutic approach is designed to inhibit C1q production in microglia. In preclinical models of neuroinflammation and demyelinating diseases like multiple sclerosis (MS), knockdown of C1qa using this ASO has been shown to reduce disease-associated microglial activation and neuroinflammatory cascades, offering a potential therapeutic strategy to prevent complement-mediated synapse loss and neurodegeneration.
- Other names
- C1q ASOC-1q ASOC 1q ASOC1qa ASOC-1qa ASOC 1qa ASOC1qa antisense oligonucleotideC-1qa antisense oligonucleotideC 1qa antisense oligonucleotide
02
Targets
C1QA (Complement C1q subcomponent subunit A)
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