Title:
METHODS FOR NUCLEIC ACID EDITING TO ALTER APOE4 FUNCTION
Document Type and Number:
WIPO Patent Application WO/2023/143609
Kind Code:
A1
Abstract:
The present application provides methods of altering ApoE4 in a cell comprising a nucleic acid encoding ApoE4 by introduction of components of a nucleic acid editing system, thereby producing an ApoE4 with altered function. Also provided are methods of ameliorating a symptom of a neurodegenerative disorder in an individual, comprising altering ApoE4 in the individual by use of a nucleic acid editing system. Also provided are ApoE4 mutants having ApoE3-like functions.
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Inventors:
YUAN PENGFEI (CN)
JIN MING (CN)
SHEN HONGYAN (CN)
ZHENG YARU (CN)
SU MEIHUA (CN)
ZHOU YONGLIN (CN)
PANG XINHE (CN)
JIN MING (CN)
SHEN HONGYAN (CN)
ZHENG YARU (CN)
SU MEIHUA (CN)
ZHOU YONGLIN (CN)
PANG XINHE (CN)
Application Number:
PCT/CN2023/073848
Publication Date:
August 03, 2023
Filing Date:
January 30, 2023
Export Citation:
Assignee:
EDIGENE THERAPEUTICS BEIJING INC (CN)
International Classes:
C07K14/47; A61P25/28; C12N15/12
Foreign References:
| CN106061510A | 2016-10-26 | |||
| CN102863525A | 2013-01-09 | |||
| US20090082271A1 | 2009-03-26 |
Other References:
TROY T ROHN ET AL.: "The Potential of CRISPR/Cas9 Gene Editing as a Treatment Strategy for Alzheimer’s Disease", 《J ALZHEIMERS DIS PARKINSONISM》, vol. 8, no. 3, 31 May 2018 (2018-05-31), XP055573399, DOI: 10.4172/2161-0460.1000439
DONG, L.-M. WEISGRABER K H: "Human apolipoprotein E4 domain interaction", JOURNAL OF BIOLOGICAL CHEMISTRY, AMERICAN SOCIETY FOR BIOCHEMISTRY AND MOLECULAR BIOLOGY, US, vol. 271, no. 32, 9 August 1996 (1996-08-09), US , pages 19053 - 19057, XP002953439, ISSN: 0021-9258, DOI: 10.1074/jbc.271.32.19053
SHENGJUN CHANG ET AL.: "Lipid- and receptor-binding regions of apolipoprotein E4 fragments act in concert to cause mitochondrial dysfunction and neurotoxicity", 《PNAS》, vol. 102, no. 51, 20 December 2005 (2005-12-20), XP002515195, DOI: 10.1073/pnas.0508254102
YUAN-TA LIN ET AL.: "APOE4 Causes Widespread Molecular and Cellular Alterations Associated with Alzheimer’s Disease Phenotypes in Human iPSC-Derived Brain Cell Types", 《NEURON》, vol. 98, 27 June 2018 (2018-06-27), XP055929562, DOI: 10.1016/j.neuron.2018.05.008
ZHONG NING, WEISGRABER KARL H.: "Understanding the Association of Apolipoprotein E4 with Alzheimer Disease: Clues from Its Structure", JOURNAL OF BIOLOGICAL CHEMISTRY, AMERICAN SOCIETY FOR BIOCHEMISTRY AND MOLECULAR BIOLOGY, US, vol. 284, no. 10, 1 March 2009 (2009-03-01), US , pages 6027 - 6031, XP093080597, ISSN: 0021-9258, DOI: 10.1074/jbc.R800009200
DONG, L.-M. WEISGRABER K H: "Human apolipoprotein E4 domain interaction", JOURNAL OF BIOLOGICAL CHEMISTRY, AMERICAN SOCIETY FOR BIOCHEMISTRY AND MOLECULAR BIOLOGY, US, vol. 271, no. 32, 9 August 1996 (1996-08-09), US , pages 19053 - 19057, XP002953439, ISSN: 0021-9258, DOI: 10.1074/jbc.271.32.19053
SHENGJUN CHANG ET AL.: "Lipid- and receptor-binding regions of apolipoprotein E4 fragments act in concert to cause mitochondrial dysfunction and neurotoxicity", 《PNAS》, vol. 102, no. 51, 20 December 2005 (2005-12-20), XP002515195, DOI: 10.1073/pnas.0508254102
YUAN-TA LIN ET AL.: "APOE4 Causes Widespread Molecular and Cellular Alterations Associated with Alzheimer’s Disease Phenotypes in Human iPSC-Derived Brain Cell Types", 《NEURON》, vol. 98, 27 June 2018 (2018-06-27), XP055929562, DOI: 10.1016/j.neuron.2018.05.008
ZHONG NING, WEISGRABER KARL H.: "Understanding the Association of Apolipoprotein E4 with Alzheimer Disease: Clues from Its Structure", JOURNAL OF BIOLOGICAL CHEMISTRY, AMERICAN SOCIETY FOR BIOCHEMISTRY AND MOLECULAR BIOLOGY, US, vol. 284, no. 10, 1 March 2009 (2009-03-01), US , pages 6027 - 6031, XP093080597, ISSN: 0021-9258, DOI: 10.1074/jbc.R800009200
Attorney, Agent or Firm:
BEIJING CAI HE LAW FIRM et al. (CN)
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