SYSTEMS AND METHODS FOR THE TREATMENT OF HEMOGLOBINOPATHIES

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.

Inventor(s): Jennifer Leah GORI, Edouard AUPEPIN DE LAMOTHE-DREUZY, Jack HEATH, John Anthony ZURIS, KaiHsin CHANG

CPC Classification: C12N15/113 (Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; {Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing (when used in plants )})

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