In vivo gene editing of tau locus via liponanoparticle delivery
The use of lipid nanoparticles specifically designed for brain delivery, encapsulating CRISPR Cas9 and gRNA, effectively addresses the challenge of editing the Tau locus in the brain, providing a novel therapeutic strategy for neurodegenerative diseases.
WO2025064591A1 Publication Date: 2025-03-27RGT UNIV OF CALIFORNIA
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Patent Information
- Application Number
- PCT/US2024/047351
- Authority / Receiving Office
- WO · WO
- Patent Type
- Applications
- Current Assignee / Owner
- Priority Date
- 2023-09-19
- Filing Date
- 2024-09-19
- Publication Date
- 2025-03-27
AI Technical Summary
Technical Problem
Current gene editing technologies face challenges in efficiently delivering nucleic acids to the brain for editing the Tau locus, which is associated with neurodegenerative diseases like Alzheimer's and Frontotemporal Dementia.
Method used
Development of a new class of lipid nanoparticles (LNPs) that are specifically configured for brain delivery, incorporating ionizable lipids, helper lipids, and cerebroside, which can encapsulate gene editing molecules like CRISPR Cas9 and gRNA to target the Tau locus.
Benefits of technology
The LNP-mediated delivery achieves efficient gene editing of the Tau locus in vivo, demonstrating higher editing frequencies compared to traditional methods, and offers a promising therapeutic approach for brain diseases.
✦ Generated by Eureka AI based on patent content.
Abstract
Lipid nanoparticles are used to deliver various types of nuclei acids to different tissues in vivo, providing a novel tool for gene editing.
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