TAM Base Editor can efficiently induce exon skipping in 42% of all DMD mutation types, reported by Professor Xing Chang at Cell Reports

Recently, the team led by Professor Xing Chang, West Lake University, published an article titled “Efficient exon skipping by base-editor-mediated abrogation of exonic splicing enhancers” in Cell Reports.

In this paper, Han Q. et al employed Targeted AID-mediated mutagenesis (TAM) cytosine base editor (CBE-TAM) to edit various hotspot mutations of DMD gene in vitro. High exon skipping efficiency can be achieved at ten exons through editing the splicing site or exonic splicing elements. This study not only proves the extensive applicability of the TAM base editor, but also brings hope to numerous DMD patients. The skipping of these ten exons skipping can target approximately 42% of all DMD mutations.

Worth noting, these results only based on in vitro study and most of them hasn’t been tested by in vivo study. More work needs to be done before they translate into true products.

In addition, Professor Chang is also the Chief Scientific Advisor of GenAssist. He is the inventor of CBE TAM. Among these mutation types, DMD exon 50 skipping drug (GEN6050) has been transferred to GenAssist and now is under IND enabling stage.

Full test link: https://www.cell.com/cell-reports/pdf/S2211-1247%2823%2901352-9.pdf