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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\u2010amino acid EcCas6e protein, derived from the Class 1 CRISPR family involved in pre\u2010crRNA processing, and conducted optimization for toxicity and editing efficiency. The ceRBE efficiently achieves both A\u2010to\u2010I and C\u2010to\u2010U base editing with low transcriptome off\u2010target in HEK293T cells. The efficient repair of the DMD Q1392X mutation (68.3\u00b110.1%) 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.<\/jats:p>","DOI":"10.1002\/advs.202206813","type":"journal-article","created":{"date-parts":[[2023,4,25]],"date-time":"2023-04-25T23:31:43Z","timestamp":1682465503000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":24,"title":["Develop a Compact RNA Base Editor by Fusing ADAR with Engineered EcCas6e"],"prefix":"10.1002","volume":"10","author":[{"given":"Xing","family":"Wang","sequence":"first","affiliation":[{"name":"HuidaGene Therapeutics Co. 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