We have developed the ACGBEmax system, which is created by fusing a dual-functional deaminase, an engineered N-methylpurine DNA glycosylase, and an evolved SRAP domain with nCas9 (D10A). This system e

NIAID Data Ecosystem30

Base editing effectively prevents the early-onset hypertrophic cardiomyopathy in Mybpc3 mutant mice

遗传性心肌病治疗

碱基编辑技术

Pathogenic variants in MYBPC3 (myosin-binding protein C3) are the leading cause of genetic hypertrophic cardiomyopathy (HCM). Currently, there is no specific and effective treatment for this disease.

NIAID Data Ecosystem20

CLUSTER guide RNAs enable precise and efficient RNA editing with endogenous ADAR enzymes in vivo

碱基编辑技术

基因治疗药物开发

RNA base editing represents a promising alternative for genome editing. Recent approaches harness the endogenous RNA editing enzyme ADAR to circumvent problems related to the ectopic expression of an

NIAID Data Ecosystem70

AAV Delivery of intein-split base editors

基因编辑递送

碱基编辑技术

These data were generated by a series of experiments designed to optimized and validate base editing in vivo. We used protein inteins to split and deliver base editors, which are too large to be deliv

NIAID Data Ecosystem30

ABE for Liver

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