Compositions and methods for gene editing

Fusion proteins with DNA methyltransferase and nuclease-deficient RNA-guided endonucleases provide a safer and more controlled method for gene silencing, addressing genotoxicity risks and achieving durable, specific gene expression regulation.

AU2026203640A1Pending Publication Date: 2026-07-23RGT UNIV OF CALIFORNIA
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Patent Information

Application Number
AU2026203640
Authority / Receiving Office
AU · AU
Patent Type
Applications
Current Assignee / Owner
Filing Date
2026-05-12
Publication Date
2026-07-23

AI Technical Summary

Technical Problem

Genome editing technologies face risks of genotoxicity from double strand breaks and an all-or-none effect on target genes, while targeted epigenome engineering offers a safer and more graded control of gene expression.

Method used

Fusion proteins comprising a DNA methyltransferase domain, XTEN linkers, and a nuclease-deficient RNA-guided endonuclease enzyme, such as CRISPR-associated proteins, are used to silence target nucleic acid sequences, allowing for controlled gene expression without the risks of genotoxicity.

Benefits of technology

The fusion proteins achieve durable, multiplexed, and highly specific silencing of target genes, reducing genotoxicity and enabling graded control of gene expression, as demonstrated by prolonged silencing and reduced viral infection rates.

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Abstract

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