Gene Correction For X-Cgd In Hematopoietic Stem And Progenitor Cells


Ontology type: sgo:Patent     


Patent Info

DATE

2022-04-21T00:00

AUTHORS

PAVEL-DINU MARA , PORTEUS MATTHEW H

ABSTRACT

The present disclosure provides methods and compositions for treating X-CGD in subjects, comprising genetically modifying cells from the subjects ex vivo by integrating a functional, codon-optimized CYBB cDNA at the endogenous CYBB locus.

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