Article
Viral delivery of compact CRISPR-Cas12f for <i>in vivo</i> gene editing applications
2024-02-09
Abstract excerpt
Treating human genetic conditions in vivo requires efficient delivery of the CRISPR gene editing machinery to the affected cells and organs. The gene editing field has seen clinical advances with ex vivo therapies and with in vivo delivery to the liver using lipid nanoparticle technology. Adeno-associated virus (AAV) serotypes have been discovered and engineered to deliver genetic material to nearly every organ...
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Identifiers and source
- Literature Corpus work
- b57b7800-3945-54eb-ae5b-9102c3d5591d
- DOI
- 10.1101/2024.02.06.578965
