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1. (WO2017152808) READ THROUGH TRUNCATED PROTEIN IN PREMATURE TERMINATION CODON DISEASES BY USING OPTIMIZED GENE CODON EXPANSION SYSTEM
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Pub. No.: WO/2017/152808 International Application No.: PCT/CN2017/075577
Publication Date: 14.09.2017 International Filing Date: 03.03.2017
IPC:
C12N 15/113 (2010.01) ,C12N 15/10 (2006.01) ,A61P 35/00 (2006.01) ,C12N 5/10 (2006.01)
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15
Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09
Recombinant DNA-technology
11
DNA or RNA fragments; Modified forms thereof
113
Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15
Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09
Recombinant DNA-technology
10
Processes for the isolation, preparation or purification of DNA or RNA
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
P
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
35
Antineoplastic agents
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
5
Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
10
Cells modified by introduction of foreign genetic material, e.g. virus-transformed cells
Applicants:
北京大学 PEKING UNIVERSITY [CN/CN]; 中国北京市 海淀区学院路38号 No. 38, Xueyuan Road, Haidian District Beijing 100191, CN
Inventors:
夏青 XIA, Qing; CN
杨琦 YANG, Qi; CN
Agent:
中国专利代理(香港)有限公司 CHINA PATENT AGENT (H.K.) LTD.; 中国香港特别行政区 湾仔港湾道23号鹰君中心22号楼 22/F, Great Eagle Centre 23 Harbour Road, Wanchai Hong Kong, CN
Priority Data:
201610134657.610.03.2016CN
Title (EN) READ THROUGH TRUNCATED PROTEIN IN PREMATURE TERMINATION CODON DISEASES BY USING OPTIMIZED GENE CODON EXPANSION SYSTEM
(FR) PROTÉINE TRONQUÉE POUR LA TRANSLECTURE DANS LES MALADIES À CODONS STOP PRÉMATURÉS EN UTILISANT UN SYSTÈME D'EXPANSION DE CODON GÉNÉTIQUE OPTIMISÉ
(ZH) 利用优化的基因密码子扩展系统通读提前终止密码子疾病中的截短蛋白
Abstract:
(EN) Provided is a method for high-efficiently reading through a nonsense mutation site in disease causing genes in monogenic hereditary diseases and recovering a normal structure and a function of a mutant protein, by using a genetic code expansion unnatural amino acid system. By modifying tRNA(tRNAPyl) of ancient methanococcus jannaschii, an all-new unnatural amino acid system that can be high-efficiently read through and that are coded by UAA and UGA, the use range of tRNAPyl and pyrrole lysyl-tRNA synthase (PylRS) orthogonal pairs is expanded. Plasmids simulating endogenous premature termination codon are constructed, so as to evaluate the read-through endogenous premature termination codon. Also provided is a system mainly comprising genes causing monogenichereditary diseases and tumor inhibitory genes in tumor cells.
(FR) L'invention concerne un procédé permettant la translecture très efficace d'un site de mutation non-sens dans une maladie responsable des gènes des maladies héréditaires monogéniques et la récupération d'une structure normale et d'une fonction d'une protéine mutée, en utilisant un système d'acides aminés non naturels à expansion de code génétique. En modifiant l'ARNt (ARNtPyl) de l'archéobactérie Methanococcus jannaschii, un tout nouveau système d'acides aminés non naturels qui peuvent être translus de manière très efficace et qui sont codés par UAA et UGA, la plage d'utilisation des paires orthogonales d'ARNtPyl et d'ARNt synthase pyrrole lysyle (PylRS) est plus importante. Des plasmides simulant un codon stop prématuré endogène sont construits de façon à évaluer la translecture du codon stop prématuré endogène. L'invention concerne également un système comprenant principalement des gènes responsables de maladies héréditaires monogéniques et des gènes inhibiteurs de tumeurs dans des cellules tumorales.
(ZH) 提供了利用基因密码扩展的非天然氨基酸系统,高效率通读单基因遗传病中致病基因的无义突变位点,恢复突变体蛋白的正常结构和功能的方法。通过改造古甲烷球菌的tRNA(tRNAPyl),得到全新的高通读效率的UAA和UGA编码的非天然氨基酸系统,扩展了tRNAPyl和吡咯赖氨酰-tRNA合成酶(PylRS)正交对的使用范围。构建了模拟内源性提前终止密码子的质粒,用于评价通读内源性提前终止密码子的效率。还提供了主要包括单基因遗传病的致病基因和肿瘤细胞内的肿瘤抑制基因的系统。
Designated States: AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KH, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW
African Regional Intellectual Property Organization (ARIPO) (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW)
Eurasian Patent Office (AM, AZ, BY, KG, KZ, RU, TJ, TM)
European Patent Office (EPO) (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, TR)
African Intellectual Property Organization (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG)
Publication Language: Chinese (ZH)
Filing Language: Chinese (ZH)