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ARTICLE | Discovery & Translation

The emerging base editor toolkit

A deep dive into how academics and biotechs are expanding the therapeutic potential of base editors by iterating on the tech’s three major types

October 20, 2023 2:31 AM UTC

Since the advent of CRISPR-Cas9 gene editing, drug developers have envisioned curing diseases by fixing gene mutations. But first-generation gene editing technologies have had limited utility due to the cuts they make in the genome, which can lead to unwanted insertions, deletions and genetic rearrangements, creating safety concerns. 

By avoiding double-strand breaks, next-generation editing tools such as base editors promise better safety, and their lightning-fast evolution and diversification is opening roads to a host of applications not previously envisioned. At the same time, the challenging capital environment is forcing the field’s leaders to prioritize applications, and at least one application of DNA base editing, CAR T creation, appears to be falling down the priority list. ...

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