Title:
MATERIALS AND METHODS FOR TREATMENT OF HEMOGLOBINOPATHIES
Document Type and Number:
WIPO Patent Application WO/2016/135557
Kind Code:
A3
Abstract:
The present application provides materials and methods for treating hemoglobinopathies. More specifically, the application provides methods for producing progenitor cells that are genetically modified via genome editing to increase the production of fetal hemoglobin (HbF), as well as modified progenitor cells (including, for example, CD34+ human hematopoietic stem cells) producing increased levels of HbF, and methods of using such cells for treating hemoglobinopathies such as sickle cell anemia and β-thalassemia.
Inventors:
PORTEUS MATTHEW HEBDEN (US)
Application Number:
PCT/IB2016/000276
Publication Date:
October 20, 2016
Filing Date:
February 23, 2016
Export Citation:
Assignee:
CRISPR THERAPEUTICS AG (CH)
International Classes:
C12N15/113; C12N15/09; C12N15/63
Domestic Patent References:
| WO2014036219A2 | 2014-03-06 | |||
| WO2013126794A1 | 2013-08-29 | |||
| WO2015006498A2 | 2015-01-15 |
Other References:
G SAGLIO ET AL: "Italian type of deletional hereditary persistence of fetal hemoglobin", BLOOD, 1 September 1986 (1986-09-01), UNITED STATES, pages 646, XP055283183, Retrieved from the Internet [retrieved on 20160623]
C CAMASCHELLA ET AL: "A new hereditary persistence of fetal hemoglobin deletion has the breakpoint within the 3' beta-globin gene enhancer", BLOOD, 15 February 1990 (1990-02-15), UNITED STATES, pages 1000, XP055283589, Retrieved from the Internet [retrieved on 20160624]
VITTORIO SEBASTIANO ET AL: "In Situ Genetic Correction of the Sickle Cell Anemia Mutation in Human Induced Pluripotent Stem Cells Using Engineered Zinc Finger Nucleases", STEM CELLS., vol. 29, no. 11, 25 October 2011 (2011-10-25), US, pages 1717 - 1726, XP055260055, ISSN: 1066-5099, DOI: 10.1002/stem.718
XIAOSONG HUANG ET AL: "Production of Gene-Corrected Adult Beta Globin Protein in Human Erythrocytes Differentiated from Patient iPSCs After Genome Editing of the Sickle Point Mutation", STEM CELLS., vol. 33, no. 5, 20 February 2015 (2015-02-20), US, pages 1470 - 1479, XP055281582, ISSN: 1066-5099, DOI: 10.1002/stem.1969
T. J. CRADICK ET AL: "CRISPR/Cas9 systems targeting -globin and CCR5 genes have substantial off-target activity", NUCLEIC ACIDS RESEARCH, vol. 41, no. 20, 1 November 2013 (2013-11-01), pages 9584 - 9592, XP055186069, ISSN: 0305-1048, DOI: 10.1093/nar/gkt714
C CAMASCHELLA ET AL: "A new hereditary persistence of fetal hemoglobin deletion has the breakpoint within the 3' beta-globin gene enhancer", BLOOD, 15 February 1990 (1990-02-15), UNITED STATES, pages 1000, XP055283589, Retrieved from the Internet
VITTORIO SEBASTIANO ET AL: "In Situ Genetic Correction of the Sickle Cell Anemia Mutation in Human Induced Pluripotent Stem Cells Using Engineered Zinc Finger Nucleases", STEM CELLS., vol. 29, no. 11, 25 October 2011 (2011-10-25), US, pages 1717 - 1726, XP055260055, ISSN: 1066-5099, DOI: 10.1002/stem.718
XIAOSONG HUANG ET AL: "Production of Gene-Corrected Adult Beta Globin Protein in Human Erythrocytes Differentiated from Patient iPSCs After Genome Editing of the Sickle Point Mutation", STEM CELLS., vol. 33, no. 5, 20 February 2015 (2015-02-20), US, pages 1470 - 1479, XP055281582, ISSN: 1066-5099, DOI: 10.1002/stem.1969
T. J. CRADICK ET AL: "CRISPR/Cas9 systems targeting -globin and CCR5 genes have substantial off-target activity", NUCLEIC ACIDS RESEARCH, vol. 41, no. 20, 1 November 2013 (2013-11-01), pages 9584 - 9592, XP055186069, ISSN: 0305-1048, DOI: 10.1093/nar/gkt714
Attorney, Agent or Firm:
PILKINGTON, Stephanie, J. (The Belgrave CentreTalbot Street, Nottingham NG1 5GG, GB)
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