Title:
SHRNA EXPRESSION CASSETTE, POLYNUCLEOTIDE SEQUENCE CARRYING SAME, AND APPLICATION THEREOF
Document Type and Number:
WIPO Patent Application WO/2018/177244
Kind Code:
A1
Abstract:
An shRNA expression cassette, a polynucleotide sequence carrying the expression cassette, and an application thereof. The shRNA expression cassette contains, in order from 5' to 3', a DNA sequence expressing the shRNA and a filler sequence. The length of the shRNA expression cassette sequence is close to the length of a wild-type AAV genome.
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Inventors:
WANG CHAO (CN)
ZHANG TINGTING (CN)
JIANG LIXIN (CN)
ZHANG TINGTING (CN)
JIANG LIXIN (CN)
Application Number:
PCT/CN2018/080480
Publication Date:
October 04, 2018
Filing Date:
March 26, 2018
Export Citation:
Assignee:
STAIDSON BEIJING BIOPHARMACEUTICALS CO LTD (CN)
BEIJING SOLOBIO GENETECHNOLOGY COMPANY LTD (CN)
BEIJING SOLOBIO GENETECHNOLOGY COMPANY LTD (CN)
International Classes:
C12N15/113; A61K31/713; A61K35/76; A61K39/23; A61K48/00; A61P9/10; A61P21/00; A61P31/18; A61P31/20; C12N5/10; C12N7/00; C12N15/864
Domestic Patent References:
| WO2005033321A2 | 2005-04-14 |
Foreign References:
| CN105612253A | 2016-05-25 | |||
| US9186419B2 | 2015-11-17 |
Other References:
ZHONG, S.M. ET AL.: "The Recombinant Adeno-associated Virus Vector (rAAV2)-mediated Apolipoprotein B mRna-specific Hammerhead Ribozyme: a Self-complementary AAV2 Vector Improves the Gene Expression", GENETIC VACCINES AND THERAPY, vol. 2, no. 1, 11 June 2004 (2004-06-11), XP021009750, ISSN: 1479-0556
See also references of EP 3613856A4
"GenBank", Database accession no. NC _001829
XIAO XIAOJUAN LIRICHARD JUDE SAMULSKI: "Production of high-titer ''recombinant adeno-associated virus vectors in the absence of helper adenovirus", J. VIROL., vol. 72, no. 3, 1998, pages 2224
GAO GPALVIRA MRWANG LCALCEDO RJOHNSTON JWILSON JM: "Novel adeno-associated viruses from rhesus monkeys as vectors for human gene theraph", PROC NATL ACAD SCI USA, vol. 99, no. 18, 3 September 2002 (2002-09-03), pages 11854 - 9
GRIEGER JCCHOI VWSAMULSKI RJ: "Production and characterization of adneo-associated viral vectors", NAT PROTOC., vol. 1, no. 3, 2006, pages 1412 - 28
WU, JZHAO, WZHONG, LHAN, ZLI, BMA, W ET AL.: "Self-complementary recombinant adeno -associated viral vectors: packaging capacity and the role of rep proteins in vector purity", HUM GENE THER., vol. 18, 2007, pages 171 - 182
JY DONGPD FANRAYMOND A, FRIZZELL: "Quantitative analysis of the packaging capacity of recombinant adeno-associated virus", HUMAN GENE THERAPY, vol. 7, 1996, pages 2101 - 2112, XP000985472
MOL THER., vol. 16, no. 5, May 2008 (2008-05-01), pages 924 - 30
See also references of EP 3613856A4
"GenBank", Database accession no. NC _001829
XIAO XIAOJUAN LIRICHARD JUDE SAMULSKI: "Production of high-titer ''recombinant adeno-associated virus vectors in the absence of helper adenovirus", J. VIROL., vol. 72, no. 3, 1998, pages 2224
GAO GPALVIRA MRWANG LCALCEDO RJOHNSTON JWILSON JM: "Novel adeno-associated viruses from rhesus monkeys as vectors for human gene theraph", PROC NATL ACAD SCI USA, vol. 99, no. 18, 3 September 2002 (2002-09-03), pages 11854 - 9
GRIEGER JCCHOI VWSAMULSKI RJ: "Production and characterization of adneo-associated viral vectors", NAT PROTOC., vol. 1, no. 3, 2006, pages 1412 - 28
WU, JZHAO, WZHONG, LHAN, ZLI, BMA, W ET AL.: "Self-complementary recombinant adeno -associated viral vectors: packaging capacity and the role of rep proteins in vector purity", HUM GENE THER., vol. 18, 2007, pages 171 - 182
JY DONGPD FANRAYMOND A, FRIZZELL: "Quantitative analysis of the packaging capacity of recombinant adeno-associated virus", HUMAN GENE THERAPY, vol. 7, 1996, pages 2101 - 2112, XP000985472
MOL THER., vol. 16, no. 5, May 2008 (2008-05-01), pages 924 - 30
Attorney, Agent or Firm:
DRAGON INTELLECTUAL PROPERTY LAW FIRM (CN)
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