Mutation-independent allele-specific crispr targeting strategy for treating genetic diseases
The mutation-independent CRISPR targeting strategy addresses the limitations of current gene therapies by targeting prevalent SNPs near disease-relevant alleles in autosomal dominant diseases, achieving effective disruption of mutant alleles and reduced disease pathology.
Patent Information
- Application Number
- PCT/CN2024/118700
- Authority / Receiving Office
- WO · WO
- Patent Type
- Applications
- Current Assignee / Owner
- Priority Date
- 2023-09-14
- Filing Date
- 2024-09-13
- Publication Date
- 2025-03-20
AI Technical Summary
Current gene-based therapeutic strategies for autosomal dominant diseases, such as Alzheimer's Disease, are limited by their focus on directly targeting disease-relevant genetic mutations, which can be ineffective due to individual variability in genetic backgrounds and the need for multiple targeting tools to cover various mutations.
A mutation-independent allele-specific CRISPR targeting strategy that targets non-disease-specific single nucleotide polymorphisms (SNPs) highly prevalent in the population, located in close proximity to disease-relevant alleles, to achieve genomic sequence disruption and inactivation of the disease-relevant allele.
This approach offers a universally applicable therapeutic option by disrupting mutant allele expression through exon excision, leading to reduced Aβ production and alleviated amyloid-related pathologies in Alzheimer's Disease, with potential for broader application in autosomal dominant disorders.