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1. (CN107849574) UTRs increasing the translation efficiency of RNA molecules

Office : China
Application Number: 201680042823.8 Application Date: 30.06.2016
Publication Number: 107849574 Publication Date: 27.03.2018
Publication Kind : A
Prior PCT appl.: Application Number:PCTEP2016065297 ; Publication Number: Click to see the data
IPC:
C12N 15/67
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
63
Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
67
General methods for enhancing the expression
CPC:
C12N 15/67
A61K 38/00
C07K 14/47
Applicants: ETHRIS GMBH
埃泽瑞斯公司
Inventors: RUDOLPH CARSTEN
C·鲁道夫
ANEJA MANISH KUMAR
M·K·阿内嘉
FERIZI MEHRIJE
M·菲瑞姿
GEIGER JOHANNES
J·盖革
Agents: 北京纪凯知识产权代理有限公司 11245
北京纪凯知识产权代理有限公司 11245
Priority Data: 15174683.1 30.06.2015 EP
Title: (EN) UTRs increasing the translation efficiency of RNA molecules
(ZH) 增加RNA分子的翻译效率的UTR
Abstract: front page image
(EN) Described is an RNA molecule comprising (a) a coding region coding for a polypeptide; and (b) upstream of the coding region one or more UTR(s) comprising the sequence as shown in SEQ ID NO:1 or a sequence which shows 1 to 4 substitutions in comparison to SEQ ID NO:1 and which results in an RNA molecule having the same or a higher translation efficiency as an RNA molecule comprising an UTR comprising SEQ ID NO:1; and/or (c) downstream of the coding region one or more UTR(s) comprising the sequence as shown in SEQ ID NO:2 or a sequence which shows 1 to 7 substitutions in comparison to SEQ ID NO:2 and which results in an RNA molecule having the same or a higher translation efficiency as an RNA molecule comprising an UTR comprising SEQ ID NO:2; wherein the polypeptide encoded by the coding region is not a cytochrome b-245 alpha polypeptide (CYBA). Moreover, described is a nucleic acid molecule encoding the RNA molecule according to the present invention. Further, described is a vector comprising the nucleic acid molecule according to the present invention and to a host cell comprising the vector according to the present invention. Further, described is a pharmaceutical composition comprising the RNA molecule according to the present invention and optionally a pharmaceutically acceptable carrier. Moreover, described is a kit comprising the RNA molecule according to the present invention. Finally, described is the use of one or more UTR(s) as defined in (b) and/or one or more UTR(s) as defined in (c) for increasing the efficiency of translating a coding region of an RNA moleculeinto a polypeptide or a protein encoded by the coding region.
(ZH) 描述了一种RNA分子,其包含(a)编码多肽的编码区;和(b)所述编码区上游的一个或多个UTR,所述UTR包含SEQ ID NO:1所示的序列或这样的序列,其与SEQ ID NO:1相比显示1至4个取代,并且其导致具有与包含UTR——包含SEQ ID NO:1——的RNA分子相同或更高的翻译效率的RNA分子;和/或(c)所述编码区下游的一个或多个UTR,所述UTR包含SEQ ID NO:2所示的序列或这样的序列,其与SEQ ID NO:2相比显示1至7个取代,并且其导致具有与包含UTR——包含SEQ ID NO:2——的RNA分子相同或更高的翻译效率的RNA分子;其中由所述编码区编码的所述多肽不是细胞色素b‑245α多肽(CYBA)。而且,描述了编码根据本发明的RNA分子的核酸分子。此外,描述了包含根据本发明的核酸分子的载体和包含根据本发明的载体的宿主细胞。此外,描述了包含根据本发明的RNA分子和任选的药学上可接受的载体的药物组合物。而且,描述了包含根据本发明的RNA分子的试剂盒。最后,描述了如(b)中所定义的一个或多个UTR和/或如(c)中所定义的一个或多个UTR用于增加将RNA分子的编码区翻译成由所述编码区编码的多肽或蛋白质的效率的用途。
Also published as:
EP3112469AU2016287436CA2990881IN201717045893KR1020180035789EP3317415
DK3112469ES2667644JP2018519830BR112017028205US20190194669WO/2017/001554