Editas Medicine, Inc.
SYSTEMS AND METHODS FOR THE TREATMENT OF HEMOGLOBINOPATHIES
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Abstract:
Genome editing systems, guide RNAs, and CRISPR-mediated methods are provided for altering portions of the HBG1 and HBG2 loci, portions of the erythroid specific enhancer of the BCL11A gene, or a combination thereof, in cells and increasing expression of fetal hemoglobin.
Status:
Application
Type:
Utility
Filling date:
27 Nov 2019
Issue date:
17 Feb 2022