Advanced Science. 2023 Apr 25
Xing Wang, Renxia Zhang, Dong Yang, Guoling Li, Zhanqing Fan, Hongting Du, Zikang Wang, Yuanhua Liu, Jiajia Lin, Xiaoqing Wu, Linyu Shi, Hui Yang, Yingsi Zhou
Products used in the paper Details Operation
AAV vector packaging AAV9 Production and Delivery to DMDQ1392X Mice: AAVs were produced by PackGene Biotech (Guangzhou, China), and applied iodixanol density gradient centrifugation for purification. Request Quote

Research Field: RNA editing

AAV Serotype: AAV9

Dose: For intramuscular injection, 8-week-old male DMDQ1392X mice were anesthetized, and the right TA (tibialis anterior) muscle was injected with 50 μL of AAV9 (5 × 10^11 vg) viral vehicles, while the left TA of the same mice were injected with same volume saline solution as a control

Routes of Administration: intramuscular injection

Animal or cell line strain: 8-week-old male DMDQ1392X mice


Catalytically inactive CRISPR-Cas13 (dCas13)-based base editors can achieve the conversion of adenine-to-inosine (A-to-I) or cytidine-to-uridine (C-to-U) at the RNA level, however, the large size of dCas13 protein limits its in vivo applications. Here, a compact and efficient RNA base editor (ceRBE) is reported with high in vivo editing efficiency. The larger dCas13 protein is replaced with a 199-amino acid EcCas6e protein, derived from the Class 1 CRISPR family involved in pre-crRNA processing, and conducted optimization for toxicity and editing efficiency. The ceRBE efficiently achieves both A-to-I and C-to-U base editing with low transcriptome off-target in HEK293T cells. The efficient repair of the DMD Q1392X mutation (68.3±10.1%) is also demonstrated in a humanized mouse model of Duchenne muscular dystrophy (DMD) after AAV delivery, achieving restoration of expression for gene products. The study supports that the compact and efficient ceRBE has great potential for treating genetic diseases.

Popular Services

AAV Packaging Service

AAV Analytical Service

Vector Design