RNA-guided gene editing and gene regulation

AU2025213563B2Pending Publication Date: 2026-09-10DUKE UNIV
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Patent Information

Application Number
AU2025213563
Authority / Receiving Office
AU · AU
Patent Type
Applications
Current Assignee / Owner
Filing Date
2025-08-05
Publication Date
2026-09-10

AI Technical Summary

Technical Problem

Existing methods for gene editing and expression manipulation, such as using zinc finger proteins and TALEs, are laborious, require specialized expertise, and face challenges in delivery and efficacy, especially for tissues like skeletal muscle, and current gene therapy strategies for diseases like Duchenne Muscular Dystrophy are limited by inefficient and unsafe delivery of therapeutic genes.

Method used

A fusion protein comprising a Cas9 protein with altered nuclease activity and a transcription activation domain, combined with guide RNA, targets specific genomic regions for precise gene modulation, including dystrophin gene correction, using systems like CRISPR/Cas9 and viral delivery.

Benefits of technology

The system enables efficient and precise modulation of gene expression and correction of mutant genes, including dystrophin, with potential for therapeutic applications in treating conditions like Duchenne Muscular Dystrophy.

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Abstract

Disclosed herein are Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) / CRISPR-associated (Cas) 9-based system related compositions and methods of using said CRISPR / Cas9-based system related compositions for altering gene expression and genome engineering. Also disclosed herein are compositions and methods of using said compositions for altering gene expression and genome engineering in muscle, such as skeletal muscle and cardiac muscle. 21962512_1 (GHMatters) P101705.AU 20 25 21 35 63 05 A ug 2 02 5 A B S T R A C T 2 0 2 5 2 1 3 5 6 3 0 5 A u g 2 0 2 5 D i s c l o s e d h e r e i n a r e C l u s t e r e d R e g u l a r l y I n t e r s p a c e d S h o r t P a l i n d r o m i c R e p e a t s ( C R I S P R ) / C R I S P R - a s s o c i a t e d ( C a s ) 9 - b a s e d s y s t e m r e l a t e d c o m p o s i t i o n s a n d m e t h o d s o f u s i n g s a i d C R I S P R / C a s 9 - b a s e d s y s t e m r e l a t e d c o m p o s i t i o n s f o r a l t e r i n g g e n e e x p r e s s i o n a n d g e n o m e e n g i n e e r i n g . A l s o d i s c l o s e d h e r e i n a r e c o m p o s i t i o n s a n d m e t h o d s o f u s i n g s a i d c o m p o s i t i o n s f o r a l t e r i n g g e n e e x p r e s s i o n a n d g e n o m e e n g i n e e r i n g i n m u s c l e , s u c h a s s k e l e t a l m u s c l e a n d c a r d i a c m u s c l e . 2 1 9 6 2 5 1 2 _ 1 ( G H M a t t e r s ) P 1 0 1 7 0 5 . A U A B S T R A C T 2 0 2 5 2 1 3 5 6 3 0 5 A u g 2 0 2 5 s k e l e t a l m u s c l e a n d c a r d i a c m u s c l e .
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Citation Information

Patent Citations

  • Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription

    WO2013176772A1