Title:
GENE THERAPY VECTOR FOR TREATING RETINITIS PIGMENTOSA DISEASE
Document Type and Number:
WIPO Patent Application WO/2020/088548
Kind Code:
A1
Abstract:
Provided is a gene therapy vector for treating a retinitis pigmentosa disease. A targeted specific optimization design is performed on a PROM1 gene coding sequence so as to obtain a nucleotide sequence particularly suitable for efficiently expressing a PROM1 protein in a mammalian (such as human) cell (in particular, a photoreceptor cell), and a recombinant AAV virus for expressing a normal human PROM1 protein is constructed. Compared with a coding sequence which is not optimized, the expression level of the PROM1 coding sequence (SEQ ID NO.:1) performed specific optimization is increased as more than three times. The sequence is particularly suitable for expressing a mammalian (such as human) cell. Eye diseases such as a retinitis pigmentosa disease can be treated efficiently.
Inventors:
LUO XUETING (CN)
SUN XIAODONG (CN)
SUN XIAODONG (CN)
Application Number:
PCT/CN2019/114446
Publication Date:
May 07, 2020
Filing Date:
October 30, 2019
Export Citation:
Assignee:
TIANJIN YUMEI PHARMATECH CO LTD (CN)
International Classes:
C12N15/12
Domestic Patent References:
| WO1991018088A1 | 1991-11-28 | |||
| WO1993009239A1 | 1993-05-13 |
Foreign References:
| JP2017121241A | 2017-07-13 | |||
| US6566118B1 | 2003-05-20 | |||
| US6989264B2 | 2006-01-24 | |||
| US6995006B2 | 2006-02-07 | |||
| US20050027091W | 2005-07-29 | |||
| US4797368A | 1989-01-10 | |||
| US6596535B1 | 2003-07-22 | |||
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| EP0488528A1 | 1992-06-03 |
Other References:
DATABASE Nucleotide 28 October 2017 (2017-10-28), S. WHORISKY: "JP 2017121241-A/59280: MODIFIED POLYNUCLEOTIDES FOR THE PRODUCTION OF PROTEINS ASSOCIATED WITH HUMAN DISEASE", XP055705612, retrieved from NCBI Database accession no. LX405239.1
DATABASE Protein 14 October 2018 (2018-10-14), Y. LEE: "prominin-1 isoform 6 precursor [Homo sapiens]", XP055705619, retrieved from NCBI Database accession no. NP_001139322
ABIGAIL T FAHIM, STEPHEN P DAIGER, RICHARD G WELEBER: "Nonsyndromic Retinitis Pigmentosa Overview", GENEREVIEWS, 19 January 2017 (2017-01-19), pages 1 - 26, XP055810223, ISSN: 2372-0697
SAMBROOK ET AL.: "Molecular Cloning: A Laboratory Manual", 1989, COLD SPRING HARBOUR LABORATORY PRESS
"Journal of Microbiology: An Experimental Handbook", PEARSON EDUCATION PRESS
See also references of EP 3875588A4
DATABASE Protein 14 October 2018 (2018-10-14), Y. LEE: "prominin-1 isoform 6 precursor [Homo sapiens]", XP055705619, retrieved from NCBI Database accession no. NP_001139322
ABIGAIL T FAHIM, STEPHEN P DAIGER, RICHARD G WELEBER: "Nonsyndromic Retinitis Pigmentosa Overview", GENEREVIEWS, 19 January 2017 (2017-01-19), pages 1 - 26, XP055810223, ISSN: 2372-0697
SAMBROOK ET AL.: "Molecular Cloning: A Laboratory Manual", 1989, COLD SPRING HARBOUR LABORATORY PRESS
"Journal of Microbiology: An Experimental Handbook", PEARSON EDUCATION PRESS
See also references of EP 3875588A4
Attorney, Agent or Firm:
XU & PARTNERS, LLC. (CN)
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