Title:
METHOD FOR INCREASING FETAL HEMOGLOBIN EXPRESSION LEVEL
Document Type and Number:
WIPO Patent Application WO/2019/080920
Kind Code:
A1
Abstract:
Provided is a method for genetic editing of an enhancer site of hematopoietic stem cell BCL11A. The genetically edited hematopoietic stem cell has the functions of a normal cell, can significantly increase the expression of fetal hemoglobin, and is used for treating β-thalassemia and sickle cell anemia.
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Inventors:
YUAN PENGFEI (CN)
FANG RIGUO (CN)
YU LINGLING (CN)
XIA PENGHUI (CN)
WANG JIA (CN)
LIANG FUCAI (CN)
FANG RIGUO (CN)
YU LINGLING (CN)
XIA PENGHUI (CN)
WANG JIA (CN)
LIANG FUCAI (CN)
Application Number:
PCT/CN2018/112068
Publication Date:
May 02, 2019
Filing Date:
October 26, 2018
Export Citation:
Assignee:
EDIGENE INC (CN)
International Classes:
C12N15/113; A61K35/18; A61P7/06; C12N5/22; C12N9/22
Domestic Patent References:
| WO2017182881A2 | 2017-10-26 | |||
| WO2013176772A1 | 2013-11-28 | |||
| WO2014065596A1 | 2014-05-01 | |||
| WO2014018423A2 | 2014-01-30 |
Foreign References:
| US20160369262A1 | 2016-12-22 | |||
| US8697359B1 | 2014-04-15 |
Other References:
WANG, YEBO: "The Optimization of CRISPR/Cas9 and Its Application in Human Stem Cells", DOCTORAL DISSERTATION, no. 6, 15 June 2017 (2017-06-15), pages 1 - 134, XP009520648, ISSN: 1674-022X
BAUER, D. E. ET AL.: "An erythroid enhancer of BCL11A subject to genetic variation determines fetal hemoglobin level", SCIENCE, vol. 342, 2013, pages 253 - 257, XP055568654, DOI: 10.1126/science.1242088
CANVER MC ET AL.: "Enhancer section by Cas9-mediated in situ saturating mutagenesis", NATURE, vol. 527, no. 7577, 12 November 2015 (2015-11-12), pages 192 - 7, XP055274680, DOI: 10.1038/nature15521
DELEEVEY GFLDAMHA MJ.: "Designing chemically modified oligonucleotides for targeted gene silencing", CHEM BIOL., vol. 19, no. 8, 24 August 2012 (2012-08-24), pages 937 - 54, XP055107150, DOI: 10.1016/j.chembiol.2012.07.011
HENDEL ET AL.: "Chemically modified guide RNAs enhance CRISPR-Cas gene editing in human primary cells", NATBIOTECHNOL., vol. 33, no. 9, September 2015 (2015-09-01), pages 985 - 989
CONG L ET AL., SCIENCE, 2013
XU ET AL., MOLECULAR THERAPY, 2017
DEWITT ET AL., SCI TRANSL MED., 2016
METTANANDA ET AL., NATURE COMMUNICATIONS, 2017
CHANG ET AL., METHODS & CLINICAL DEVELOPMENT, 2017
MATTEW C ET AL., NATURE, 2015
ANTONIO CAO ET AL., GENES IN MEDICINE, 2010
GUIDELINES FOR DIAGNOSIS AND TREATMENT OF SEVERE B THALASSEMIA, 2010
LIN YE ET AL., PNAS, 2016
MATTHEW H. PORTEUS, ADVANCES IN EXPERIMENTAL MEDICINE AND BIOLOGY, 2013
BAUER, D. E. ET AL.: "An erythroid enhancer of BCL11A subject to genetic variation determines fetal hemoglobin level", SCIENCE, vol. 342, 2013, pages 253 - 257, XP055568654, DOI: 10.1126/science.1242088
CANVER MC ET AL.: "Enhancer section by Cas9-mediated in situ saturating mutagenesis", NATURE, vol. 527, no. 7577, 12 November 2015 (2015-11-12), pages 192 - 7, XP055274680, DOI: 10.1038/nature15521
DELEEVEY GFLDAMHA MJ.: "Designing chemically modified oligonucleotides for targeted gene silencing", CHEM BIOL., vol. 19, no. 8, 24 August 2012 (2012-08-24), pages 937 - 54, XP055107150, DOI: 10.1016/j.chembiol.2012.07.011
HENDEL ET AL.: "Chemically modified guide RNAs enhance CRISPR-Cas gene editing in human primary cells", NATBIOTECHNOL., vol. 33, no. 9, September 2015 (2015-09-01), pages 985 - 989
CONG L ET AL., SCIENCE, 2013
XU ET AL., MOLECULAR THERAPY, 2017
DEWITT ET AL., SCI TRANSL MED., 2016
METTANANDA ET AL., NATURE COMMUNICATIONS, 2017
CHANG ET AL., METHODS & CLINICAL DEVELOPMENT, 2017
MATTEW C ET AL., NATURE, 2015
ANTONIO CAO ET AL., GENES IN MEDICINE, 2010
GUIDELINES FOR DIAGNOSIS AND TREATMENT OF SEVERE B THALASSEMIA, 2010
LIN YE ET AL., PNAS, 2016
MATTHEW H. PORTEUS, ADVANCES IN EXPERIMENTAL MEDICINE AND BIOLOGY, 2013
Attorney, Agent or Firm:
BEIJING CAI HE LAW FIRM (CN)
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