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AssigneeEmendoBio Inc.
InventorsDavid Baram, Lior Izhar, Asael Herman, Rafi Emmanuel, Liat Rockah, Nadav Marbach-Bar, Michal Golan Mashiach, Joseph Georgeson
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Methods for inactivating in a cell a mutant allele of the elastase, neutrophil expressed gene (ELANE gene) gene having a mutation associated with severe congenital neutropenia (SCN) or cyclic neutropenia (CyN) wherein the mutant allele is selected from the group consisting of the ELANE mutants set forth in Table 1, the method comprising introducing to the cell a composition comprising: a CRISPR nuclease or a sequence encoding the CRISPR nuclease; and a first RNA molecule comprising a guide sequence portion having 17-30 nucleotides, wherein a complex of the CRISPR nuclease and the first RNA molecule affects a double strand break in the mutant allele of the ELANE gene the method optionally further comprising introduction of a second RNA molecule comprising a guide sequence portion capable of complexing with a CRISPR nuclease, wherein the complex of the second RNA molecule and CRISPR nuclease affects a second double strand break in the ELANE gene.
An ex vivo or in vitro method of modifying in a cell a mutant allele of the elastase, neutrophil expressed (ELANE) gene wherein the mutant allele has a mutation associated with severe congenital neutropenia (SCN) or cyclic neutropenia (CyN), the method comprising introducing to the cell a composition comprising:
Dependencies are inferred from the claim text.