Title:
COMPOSITIONS AND METHODS FOR TREATMENT OF TRANSTHYRETIN AMYLOIDOSIS
Document Type and Number:
WIPO Patent Application WO/2023/185697
Kind Code:
A2
Abstract:
Compositions and methods are described herein for treating subjects having amyloidosis associated with transthyretin (ATTR).
Inventors:
SHI FUXIN (US)
CAO WENHU (CN)
CHEN YE (CN)
ZHANG PENGCHENG (CN)
KHATOON NAFEESA (CN)
LIU HUANLE (CN)
CAO WENHU (CN)
CHEN YE (CN)
ZHANG PENGCHENG (CN)
KHATOON NAFEESA (CN)
LIU HUANLE (CN)
Application Number:
PCT/CN2023/083904
Publication Date:
October 05, 2023
Filing Date:
March 24, 2023
Export Citation:
Assignee:
ACCUREDIT THERAPEUTICS SUZHOU CO LTD (CN)
International Classes:
C12N15/113; A61K31/713
Domestic Patent References:
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| WO2020198697A1 | 2020-10-01 | |||
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MAKAROVA ET AL., NAT. REV. MICROBIOL, vol. 13, 2015, pages 722 - 36
YIN J ET AL.: "Cas9 exo-endonuclease eliminates chromosomal translocations during genome editing", NAT COMMUN., vol. 13, no. 1, 8 March 2022 (2022-03-08), pages 1204
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SAMBROOK: "Molecular Cloning: A Laboratory Manual", 1989
GAIT, OLIGONUCLEOTIDE SYNTHESIS, 1984
FRESHNEY, ANIMAL CELL CULTURE, 1987
WEIR: "Handbook of Experimental Immunology", METHODS IN ENZYMOLOGY, 1996
MILLERCALOS, GENE TRANSFER VECTORS FOR MAMMALIAN CELLS, 1987
AUSUBEL, CURRENT PROTOCOLS IN MOLECULAR BIOLOGY, 1987
MULLIS, PCR: THE POLYMERASE CHAIN REACTION, 1994
COLIGAN, CURRENT PROTOCOLS IN IMMUNOLOGY, 1991
TSAI ET AL., NATURE BIOTECHNOLOGY, vol. 33, 2015, pages 187 - 197
YIN ET AL., CELL DISCOVERY, vol. 5, no. 18, 2019, pages 18
YIN, J.LU, C. ET AL., NAT COMMUN, vol. 13, 2022, pages 1204
GILAD A. ET AL., JACC, vol. 77, 11 May 2021 (2021-05-11), pages 18
"The Biochemistry of the Nucleic Acids 5-36", 1992
VESTERWENGEL, BIOCHEMISTRY, vol. 43, no. 42, 2004, pages 13233 - 41
ZETSCHE ET AL., CELL, vol. 163, 2015, pages 1 - 13
MAKAROVA ET AL., NAT REV MICROBIOL, vol. 13, no. 11, 2015, pages 722 - 36
SHMAKOV ET AL., MOLECULAR CELL, vol. 60, 2015, pages 385 - 397
"NCBI", Database accession no. 7276
MAKAROVA ET AL., NAT. REV. MICROBIOL., vol. 9, 2011, pages 467 - 477
MAKAROVA ET AL., NAT. REV. MICROBIOL, vol. 13, 2015, pages 722 - 36
YIN J ET AL.: "Cas9 exo-endonuclease eliminates chromosomal translocations during genome editing", NAT COMMUN., vol. 13, no. 1, 8 March 2022 (2022-03-08), pages 1204
SCHMIDT ET AL., NATURE METHODS, vol. 4, 2007, pages 1051 - 1057
SHENDURE ET AL., NATURE, vol. 26, 2008, pages 1135 - 1145
HOTTA M ET AL., RADIOGRAPHICS, vol. 40, 2020, pages 2029 - 2041
MULLER ML ET AL., EUROPEAN JOURNAL OF HEART FAILURE, vol. 22, 2020, pages 39 - 53
SAMBROOK: "Molecular Cloning: A Laboratory Manual", 1989
GAIT, OLIGONUCLEOTIDE SYNTHESIS, 1984
FRESHNEY, ANIMAL CELL CULTURE, 1987
WEIR: "Handbook of Experimental Immunology", METHODS IN ENZYMOLOGY, 1996
MILLERCALOS, GENE TRANSFER VECTORS FOR MAMMALIAN CELLS, 1987
AUSUBEL, CURRENT PROTOCOLS IN MOLECULAR BIOLOGY, 1987
MULLIS, PCR: THE POLYMERASE CHAIN REACTION, 1994
COLIGAN, CURRENT PROTOCOLS IN IMMUNOLOGY, 1991
TSAI ET AL., NATURE BIOTECHNOLOGY, vol. 33, 2015, pages 187 - 197
YIN ET AL., CELL DISCOVERY, vol. 5, no. 18, 2019, pages 18
YIN, J.LU, C. ET AL., NAT COMMUN, vol. 13, 2022, pages 1204
Attorney, Agent or Firm:
BEIJING YINGKE LAW FIRM (CN)
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