CRISPR/Cas Genome Editing for Aggregation-Linked Neurodegeneration: Opportunity and Translational Constraint

Abstract

A critical review of CRISPR/Cas approaches for modeling and potentially modifying aggregation-linked neurodegenerative disease. It centers delivery, mosaicism, off-target effects, immune response, irreversibility, and uncertain clinical endpoints.

Educational notice: This abstract is for scientific education only. It is not medical advice, diagnosis, or treatment guidance.