CRISPR–Cas13 systems have been developed for precise RNA editing, and can potentially be used therapeutically when temporary changes are desirable or when DNA editing is challenging. We have identified and characterized an ultrasmall family of Cas13b proteins—Cas13bt—that can mediate mammalian transcript knockdown. We have engineered compact variants of REPAIR and RESCUE RNA editors by functionalizing Cas13bt with adenosine and cytosine deaminase domains, and demonstrated packaging of the editors within a single adeno-associated virus.

中文翻译：

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带有小 Cas13 蛋白的紧凑型 RNA 编辑器

CRISPR–Cas13 系统已被开发用于精确的 RNA 编辑，并且在需要临时改变或 DNA 编辑具有挑战性时可能用于治疗。我们已经鉴定并表征了一个超小型的 Cas13b 蛋白家族——Cas13bt——它可以介导哺乳动物转录物的敲除。我们通过使用腺苷和胞嘧啶脱氨酶结构域对 Cas13bt 进行功能化，设计了 REPAIR 和 RESCUE RNA 编辑器的紧凑变体，并展示了编辑器在单个腺相关病毒中的包装。