Published in

American Association for the Advancement of Science, Science Advances, 43(6), 2020

DOI: 10.1126/sciadv.abb7107

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Supramolecular nanosubstrate–mediated delivery system enables CRISPR-Cas9 knockin of hemoglobin beta gene for hemoglobinopathies

This paper is made freely available by the publisher.
This paper is made freely available by the publisher.

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Abstract

CRISPR-Cas9 knockin of the HBB gene paves the way for a general therapeutic solution for treating hemoglobinopathies.