Title:
DIFFERENTIAL KNOCKOUT OF AN ALLELE OF A HETEROZYGOUS APOLIPOPROTEIN A1 (APOA1) GENE
Document Type and Number:
WIPO Patent Application WO/2019/190930
Kind Code:
A3
Abstract:
RNA molecules comprising a guide sequence portion having 17-20 nucleotides in the sequence of 17-20 contiguous nucleotides set forth in any one of SEQ ID NOs: 1-1313 and compositions, methods, and uses thereof.
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Inventors:
BARAM DAVID (IL)
IZHAR LIOR (IL)
HERMAN ASAEL (IL)
EMMANUEL RAFI (IL)
GOLAN MASHIACH MICHAL (IL)
GEORGESON JOSEPH (IL)
IZHAR LIOR (IL)
HERMAN ASAEL (IL)
EMMANUEL RAFI (IL)
GOLAN MASHIACH MICHAL (IL)
GEORGESON JOSEPH (IL)
Application Number:
PCT/US2019/023715
Publication Date:
November 07, 2019
Filing Date:
March 22, 2019
Export Citation:
Assignee:
EMENDOBIO INC (US)
BARAM DAVID (IL)
IZHAR LIOR (IL)
HERMAN ASAEL (IL)
EMMANUEL RAFI (IL)
GOLAN MASHIACH MICHAL (IL)
GEORGESON JOSEPH (IL)
BARAM DAVID (IL)
IZHAR LIOR (IL)
HERMAN ASAEL (IL)
EMMANUEL RAFI (IL)
GOLAN MASHIACH MICHAL (IL)
GEORGESON JOSEPH (IL)
International Classes:
C12N9/22; C12Q1/68; C12N15/11; C12N15/85
Foreign References:
| JP2011022146A | 2011-02-03 | |||
| US20050172359A1 | 2005-08-04 | |||
| US20100168006A1 | 2010-07-01 |
Other References:
MONTEYS, AM ET AL.: "CRISPR/Cas9 Editing of the Mutant Huntingtin Allele In Vitro and In Vivo", MOLECULAR THERAPY, vol. 25, no. 1, 4 January 2017 (2017-01-04), pages 12 - 23, XP055574814
ERIKSSON, M ET AL.: "Hereditary Apolipoprotein Al-Associated Amyloidosis in Surgical Pathology Specimens: Identification of Three Novel Mutations in the APOA1 Gene", THE JOURNAL OF MOLECULAR DIAGNOSTICS, vol. 11, no. 3, 26 March 2009 (2009-03-26), pages 257 - 262, XP055650600, DOI: 10.2353/jmoldx.2009.080161
DELGRADO-LISTA, J ET AL.: "Effects of Variations in the Apoa1/C3/A4/A5 Gene Cluster on Different Parameters of Postprandial Lipid Metabolism in Healthy Young Men", JOURNAL OF LIPID RESEARCH, vol. 51, no. 1, January 2010 (2010-01-01), pages 63 - 73, XP055650607, DOI: 10.1194/jlr.M800527-JLR200
ERIKSSON, M ET AL.: "Hereditary Apolipoprotein Al-Associated Amyloidosis in Surgical Pathology Specimens: Identification of Three Novel Mutations in the APOA1 Gene", THE JOURNAL OF MOLECULAR DIAGNOSTICS, vol. 11, no. 3, 26 March 2009 (2009-03-26), pages 257 - 262, XP055650600, DOI: 10.2353/jmoldx.2009.080161
DELGRADO-LISTA, J ET AL.: "Effects of Variations in the Apoa1/C3/A4/A5 Gene Cluster on Different Parameters of Postprandial Lipid Metabolism in Healthy Young Men", JOURNAL OF LIPID RESEARCH, vol. 51, no. 1, January 2010 (2010-01-01), pages 63 - 73, XP055650607, DOI: 10.1194/jlr.M800527-JLR200
Attorney, Agent or Firm:
GERSHIK, Gary, J. (US)
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