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Research paper · Foundational
Multiplex genome engineering using CRISPR/Cas systems
One of the two January 2013 papers showing that CRISPR works inside human cells.
Simple explanation
Working in a test tube is one thing; working inside a living human cell, with its packed chromosomes and defence systems, is another. This paper showed CRISPR editing human and mouse cells, and doing several edits at once.
Go deeper
Cong, Ran, Cox, Lin, Barretto, Habib, Hsu, Wu, Jiang, Marraffini and Zhang demonstrated CRISPR-Cas9 genome editing in human and mouse cells, including multiplexed editing at several loci simultaneously and use of a Cas9 nickase to reduce off-target activity.
Educational information only
This page is a reference, not medical advice. Research and regulatory status change; check the last-updated date above and confirm anything important against the primary sources listed.