Gene expression of deep intron mutations

摘要

Composition for treating diseases or disorders associated with deep intron mutations using a genetically engineered, non-naturally occurring Clustered Regular Interspaced Short Palindromatic Repeats (CRISPR) -CRISPR-related (Cas) (CRISPR-Cas) system Articles, methods, kits and viral particles are provided herein. In some aspects, a self-limiting CRISPER-Cas system is provided herein.