COMPOSITIONS AND METHODS FOR THE TARGETING OF BCL11A

Provided herein are systems comprising Class 2, Type V CRISPR polypeptides, guide nucleic acids (gNA), and optionally donor template nucleic acids useful in the modification of a BCL11A gene. The systems are also useful for the modification of cells in subjects with a hemoglobinopathy-related diseas...

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Hauptverfasser:	STAAHL, Brett T, DENNY, Sarah, URNES, Cole, COLIN, Isabel, ADIL, Maroof, HIGGINS, Sean, OAKES, Benjamin
Format:	Patent
Sprache:	eng ; fre
Online-Zugang:	Volltext bestellen
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