Antisense oligonucleotides targeting EZH2 splicing and their use for treating cancer and kidney disease
Antisense oligonucleotides (ASOs) that promote exon 14 skipping of the EZH2 gene provide a novel therapeutic approach for treating HCC and other conditions by effectively downregulating H3K27me3 expression, leading to improved treatment outcomes.
Patent Information
- Application Number
- PCT/CN2024/122452
- Authority / Receiving Office
- WO · WO
- Patent Type
- Applications
- Current Assignee / Owner
- Priority Date
- 2023-09-29
- Filing Date
- 2024-09-29
- Publication Date
- 2025-04-03
AI Technical Summary
Current treatments for hepatocellular carcinoma (HCC) and other conditions such as chronic kidney disease, acute kidney injury, neurodegeneration in ataxia-telangiectasia, retinal degeneration, and inflammation lack effectiveness due to limited therapeutic options and resistance to existing therapies, highlighting the need for novel molecular approaches targeting EZH2 splicing.
Development of antisense oligonucleotides (ASOs) that specifically promote exon 14 skipping of the EZH2 gene, thereby downregulating H3K27me3 expression, which can be used to treat various conditions by administering these ASOs alone or via expression vectors.
The use of ASOs to induce exon 14 skipping in the EZH2 gene effectively downregulates H3K27me3 expression, leading to significant inhibition of tumor growth, promotion of apoptosis, and improvement of renal function in both cancer and kidney disease models, thereby offering a promising therapeutic strategy.