ABE-ultramax for high-efficiency biallelic adenine base editing in zebrafish

Wei Qin, Fang Liang, Sheng-Jia Lin, Cassidy Petree, Kevin Huang, Yu Zhang, Lin Li, Pratishtha Varshney, Philippe Mourrain, Yanmei Liu () and Gaurav K. Varshney ()
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Wei Qin: Oklahoma Medical Research Foundation
Fang Liang: South China Normal University
Sheng-Jia Lin: Oklahoma Medical Research Foundation
Cassidy Petree: Oklahoma Medical Research Foundation
Kevin Huang: Oklahoma Medical Research Foundation
Yu Zhang: Oklahoma Medical Research Foundation
Lin Li: South China Normal University
Pratishtha Varshney: Oklahoma Medical Research Foundation
Philippe Mourrain: Stanford University
Yanmei Liu: South China Normal University
Gaurav K. Varshney: Oklahoma Medical Research Foundation

Nature Communications, 2024, vol. 15, issue 1, 1-14

Abstract: Abstract Advancements in CRISPR technology, particularly the development of base editors, revolutionize genetic variant research. When combined with model organisms like zebrafish, base editors significantly accelerate and refine in vivo analysis of genetic variations. However, base editors are restricted by protospacer adjacent motif (PAM) sequences and specific editing windows, hindering their applicability to a broad spectrum of genetic variants. Additionally, base editors can introduce unintended mutations and often exhibit reduced efficiency in living organisms compared to cultured cell lines. Here, we engineer a suite of adenine base editors (ABEs) called ABE-Ultramax (Umax), demonstrating high editing efficiency and low rates of insertions and deletions (indels) in zebrafish. The ABE-Umax suite of editors includes ABEs with shifted, narrowed, or broadened editing windows, reduced bystander mutation frequency, and highly flexible PAM sequence requirements. These advancements have the potential to address previous challenges in disease modeling and advance gene therapy applications.

Date: 2024
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DOI: 10.1038/s41467-024-49943-1

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