Title:
MRNA MOLECULE ENCODING ALDH2 POLYPEPTIDE, USE THEREOF, AND MRNA DRUG
Document Type and Number:
WIPO Patent Application WO/2023/246354
Kind Code:
A1
Abstract:
Provided are an mRNA molecule encoding ALDH2 polypeptide, use thereof, and an mRNA drug. In some embodiments, an mRNA molecule or an mRNA drug comprising an open reading frame encoding ALDH2 enzyme is provided. The open reading frame is sequence-optimized. In specific embodiments, sequence-optimized polynucleotides comprising nucleotides encoding a polypeptide sequence of human ALDH2, or sequences having a high sequence identity to the sequence-optimized polynucleotides are provided.
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Inventors:
PENG YUCAI (CA)
ZHANG ZHENDONG (CN)
FANG ZIHUI (CN)
LIU JUN (CN)
LIU QI (CN)
LUO LIPING (CN)
LEI YIXIN (CN)
LI SHUANG (CN)
ZHANG ZHENDONG (CN)
FANG ZIHUI (CN)
LIU JUN (CN)
LIU QI (CN)
LUO LIPING (CN)
LEI YIXIN (CN)
LI SHUANG (CN)
Application Number:
PCT/CN2023/093362
Publication Date:
December 28, 2023
Filing Date:
May 11, 2023
Export Citation:
Assignee:
LIVERNA THERAPEUTICS INC (CN)
International Classes:
A61K48/00; C12N15/53; A61P3/00; C12N15/63
Domestic Patent References:
| WO2019195592A1 | 2019-10-10 |
Foreign References:
| CN114891808A | 2022-08-12 | |||
| US20190160187A1 | 2019-05-30 |
Other References:
HU XIUYING; FANG QIN; WANG JISHI; MA DAN; JIANG LI: "A Preliminary Study of Improving the Function of Vascular Endothelial Cells with High Expression of ALDH2 Gene", JOURNAL OF GUIYANG MEDICAL COLLEGE, GUIZHOU MEDICAL UNIVERSITY, CN, vol. 41, no. 11, 30 November 2016 (2016-11-30), CN , pages 1263 - 1268, XP009551589, ISSN: 1000-2707, DOI: 10.19367/j.cnki.1000-2707.2016.11.006
MATSUMURA, Y. ET AL.: "Gene Therapy Correction of Aldehyde Dehydrogenase 2 Deficiency", MOLECULAR THERAPY: METHODS & CLINICAL DEVELOPMENT, vol. 15, 31 December 2019 (2019-12-31), XP093112692, DOI: 10.1016/j.omtm.2019.08.004
MATSUMURA YUKI, LI NA, ALWASEEM HANAN, PAGOVICH ODELYA E., CRYSTAL RONALD G., GREENBLATT MATTHEW B., STILES KATIE M.: "Systemic Adeno-Associated Virus-Mediated Gene Therapy Prevents the Multiorgan Disorders Associated with Aldehyde Dehydrogenase 2 Deficiency and Chronic Ethanol Ingestion", HUMAN GENE THERAPY, MARY ANN LIEBERT, INC. PUBLISHERS, GB, vol. 31, no. 3-4, 1 February 2020 (2020-02-01), GB , pages 163 - 182, XP093121393, ISSN: 1043-0342, DOI: 10.1089/hum.2019.268
QIN SHUGANG, TANG XIAOSHAN, CHEN YUTING, CHEN KEPAN, FAN NA, XIAO WEN, ZHENG QIAN, LI GUOHONG, TENG YUQING, WU MIN, SONG XIANGRONG: "mRNA-based therapeutics: powerful and versatile tools to combat diseases", SIGNAL TRANSDUCTION AND TARGETED THERAPY, vol. 7, no. 1, XP093096023, DOI: 10.1038/s41392-022-01007-w
MATSUMURA, Y. ET AL.: "Gene Therapy Correction of Aldehyde Dehydrogenase 2 Deficiency", MOLECULAR THERAPY: METHODS & CLINICAL DEVELOPMENT, vol. 15, 31 December 2019 (2019-12-31), XP093112692, DOI: 10.1016/j.omtm.2019.08.004
MATSUMURA YUKI, LI NA, ALWASEEM HANAN, PAGOVICH ODELYA E., CRYSTAL RONALD G., GREENBLATT MATTHEW B., STILES KATIE M.: "Systemic Adeno-Associated Virus-Mediated Gene Therapy Prevents the Multiorgan Disorders Associated with Aldehyde Dehydrogenase 2 Deficiency and Chronic Ethanol Ingestion", HUMAN GENE THERAPY, MARY ANN LIEBERT, INC. PUBLISHERS, GB, vol. 31, no. 3-4, 1 February 2020 (2020-02-01), GB , pages 163 - 182, XP093121393, ISSN: 1043-0342, DOI: 10.1089/hum.2019.268
QIN SHUGANG, TANG XIAOSHAN, CHEN YUTING, CHEN KEPAN, FAN NA, XIAO WEN, ZHENG QIAN, LI GUOHONG, TENG YUQING, WU MIN, SONG XIANGRONG: "mRNA-based therapeutics: powerful and versatile tools to combat diseases", SIGNAL TRANSDUCTION AND TARGETED THERAPY, vol. 7, no. 1, XP093096023, DOI: 10.1038/s41392-022-01007-w
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