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1. US20210363547 - DIFFERENTIAL KNOCKOUT OF AN ALLELE OF A HETEROZYGOUS ELANE GENE

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United States of America
Application Number 17053629
Application Date 06.05.2019
Publication Number 20210363547
Publication Date 25.11.2021
Publication Kind A1
IPC
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90Stable introduction of foreign DNA into chromosome
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Applicants David Baram
EMENDOBIO INC.
Lior Izhar
Asael Herman
Rafi Emmanuel
Michal Golan Mashiach
Joseph Georgeson
Inventors David Baram
Lior Izhar
Asael Herman
Rafi Emmanuel
Michal Golan Mashiach
Joseph Georgeson
Title
(EN) DIFFERENTIAL KNOCKOUT OF AN ALLELE OF A HETEROZYGOUS ELANE GENE
Abstract
(EN)

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) and which cell is heterozygous at one or more polymorphic sites selected from the group consisting of: rs10414837, rs3761005, rs1683564, rs9749274, rs740021, rs201048029, rs199720952, rs28591229, rs71335276, rs58082177, rs3826946, rs10413889, rs761481944, rs3761008, rs10409474, rs3761007, rs17216649, rs10469327, rs8107095, rs10424470 and rs78302854, 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 17-20 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.


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