Chapter 3 —— 37 —— off-target effects, including gRNA-independent deamination, will complement the safety profile of DMD-targeting ABE:gRNA complexes. Concluding, Chai and colleagues demonstrate that AAV-vectored ABE transsplicing can induce robust synthesis of Becker-like dystrophins in striated muscles of dystrophic mice upon DSB-free splice site knockout and exon skipping [5], The resulting improvement in pathological traits measured at the molecular, tissue, and functional levels validates the use of this platform for the efficacious testing and optimization of the growing number of ABE reagents in vivo and expands the range of potential treatment modalities for DMD patients. Acknowledgements Research in our laboratory is supported by the Prinses Beatrix Spierfonds (W.OR21-01), the Duchenne Parent Project NL, the Dutch Research Council (NWO) – Open Technology Program, and EU Marie Skłodowska-Curie Doctoral Network Actions. Z.L. holds a Ph.D. fellowship from the China Scholarship Council – Leiden University Joint Scholarship Program. Authors of this work are members of the European Reference Network – Neuromuscular diseases (ERN EURO-NMD). Declaration of interest The authors declare no competing interests.
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