公开(公告)号：CN106497807A

公开(公告)日：2017-03-15

申请号：CN201610928565.5

申请日：2016-10-31

Applicant: 江南大学

Inventor： 吴殿辉 ,  陆健 ,  李晓敏 ,  谢广发 ,  孙军勇 ,  蔡国林

IPC: C12N1/19 ,  C12N15/81 ,  C12G3/02 ,  C12R1/865

CPC classification number: C12N9/1018 ,  C12G3/02 ,  C12Y201/03003

Abstract: 本发明公开了一种低产瓜氨酸的工业酿酒酵母代谢工程菌，属于发酵和生物技术领域。本发明以酿酒酵母尿嘧啶缺陷型的恢复为筛选标记，构建鸟氨酸氨甲酰转移酶的编码基因ARG3的敲除组件ARG3L-URA3-ARG3R，转化至尿嘧啶缺陷型单倍体，通过同源重组在酿酒酵母基因组ARG3的开放阅读框插入完整的酿酒酵母URA3基因，获得敲除了ARG3基因的单倍体工程菌。利用该方法获得的酵母工程菌为原养型菌株，不含外源基因，具有生物安全性，与出发菌株在米汁发酵过程中生长和发酵性能相似，但瓜氨酸的含量降低了39.0％，为工业化生产中降低EC含量提供了新思路。

公开(公告)号：CN103748078B

公开(公告)日：2016-11-09

申请号：CN201280036307.6

申请日：2012-06-08

Applicant: 夏尔人类遗传性治疗公司

Inventor： M·哈特莱因 ,  B·C·吉尔德 ,  F·德罗莎 ,  J·C·张

IPC: C07D233/64 ,  C07C323/00 ,  A61K9/127 ,  A61K31/4164

CPC classification number: A61K9/19 ,  A61K9/1271 ,  A61K9/1272 ,  A61K9/5123 ,  A61K31/7088 ,  A61K38/45 ,  A61K38/465 ,  A61K38/47 ,  A61K38/50 ,  C07C323/25 ,  C07C323/27 ,  C07C323/44 ,  C07D233/64 ,  C07J41/0055 ,  C07J43/003 ,  C12N15/88 ,  C12Y201/03003 ,  C12Y203/01 ,  C12Y207/08015 ,  C12Y301/06 ,  C12Y302/01022 ,  C12Y302/01035 ,  C12Y302/01046 ,  C12Y302/01076 ,  C12Y305/03001

Abstract: 本文所公开的是新型化合物、包括此类化合物的药物组合物及其使用的相关方法。本文所述的化合物用作如脂质体递送媒介物以促进包封的多核苷酸向靶细胞的递送以及所述靶细胞随后的转染，并且在某些实施方案中所述化合物的特征在于具有一种或多种性质，所述性质提供此类化合物相对于其它类似类别的脂质的优点。

公开(公告)号：CN103282486B

公开(公告)日：2016-02-17

申请号：CN201180064409.4

申请日：2011-12-08

Applicant: CJ第一制糖株式会社

Inventor： 崔香 ,  李京珉 ,  姜玟先 ,  全成后 ,  严惠媛 ,  崔秀真 ,  李翰元 ,  申秀安

IPC: C12N1/21 ,  C12N15/74 ,  C12P13/10 ,  C12R1/15

CPC classification number: C12P13/10 ,  C07K14/34 ,  C12N9/0008 ,  C12N9/1018 ,  C12N9/1029 ,  C12N9/1096 ,  C12N9/1217 ,  C12R1/15 ,  C12Y102/01038 ,  C12Y201/03003 ,  C12Y203/01035 ,  C12Y206/01013 ,  C12Y207/02008

Abstract: 本发明涉及一种具有改善的鸟氨酸生产能力的微生物，其中自鸟氨酸至精氨酸的生物合成途径被阻断，细胞内的谷氨酸水平增加，并且自谷氨酸至鸟氨酸的生物合成途径被增强，并且本发明涉及一种使用该微生物生产鸟氨酸的方法。

公开(公告)号：EP2650358B1

公开(公告)日：2018-10-17

申请号：EP11846603.6

申请日：2011-12-08

Applicant: CJ CheilJedang Corporation

Inventor： CHOI, Hyang ,  LEE, Kyoung Min ,  KANG, Min Sun ,  JHON, Sung Hoo ,  UM, Hye Won ,  CHOI, Su Jin ,  LEE, Han Won ,  SHIN, Soo An

IPC: C12N1/21 ,  C12N15/74 ,  C12P13/10 ,  C12R1/15

CPC classification number: C12P13/10 ,  C07K14/34 ,  C12N9/0008 ,  C12N9/1018 ,  C12N9/1029 ,  C12N9/1096 ,  C12N9/1217 ,  C12R1/15 ,  C12Y102/01038 ,  C12Y201/03003 ,  C12Y203/01035 ,  C12Y206/01013 ,  C12Y207/02008

Abstract: The present invention relates to a microorganism having an improved ornithine-producing ability, in which the biosynthetic pathway of arginine from ornithine is blocked, the intracellular glutamate level is increased, and the biosynthetic pathway of ornithine from glutamate is enhanced, and a method for producing ornithine using the microorganism.

公开(公告)号：EP2650358A2

公开(公告)日：2013-10-16

申请号：EP11846603.6

申请日：2011-12-08

Applicant: CJ CheilJedang Corporation

Inventor： CHOI, Hyang ,  LEE, Kyoung Min ,  KANG, Min Sun ,  JHON, Sung Hoo ,  UM, Hye Won ,  CHOI, Su Jin ,  LEE, Han Won ,  SHIN, Soo An

IPC: C12N1/21 ,  C12N15/74 ,  C12P13/10 ,  C12R1/15

CPC classification number: C12P13/10 ,  C07K14/34 ,  C12N9/0008 ,  C12N9/1018 ,  C12N9/1029 ,  C12N9/1096 ,  C12N9/1217 ,  C12R1/15 ,  C12Y102/01038 ,  C12Y201/03003 ,  C12Y203/01035 ,  C12Y206/01013 ,  C12Y207/02008

Abstract: The present invention relates to a microorganism having an improved ornithine-producing ability, in which the biosynthetic pathway of arginine from ornithine is blocked, the intracellular glutamate level is increased, and the biosynthetic pathway of ornithine from glutamate is enhanced, and a method for producing ornithine using the microorganism.

公开(公告)号：EP3212793A4

公开(公告)日：2018-05-16

申请号：EP15855964

申请日：2015-11-01

Applicant: ARCTURUS THERAPEUTICS INC

Inventor： CHIVUKULA PADMANABH ,  WARREN LUIGI ,  TACHIKAWA KIYOSHI ,  PAYNE JOSEPH E

IPC: C12N15/11 ,  A61K39/00 ,  A61K48/00 ,  C07K14/445 ,  C07K14/46 ,  C07K14/47 ,  C07K14/505 ,  C07K14/52 ,  C07K14/575 ,  C07K14/705 ,  C07K14/745 ,  C07K14/805 ,  C07K14/81 ,  C12N9/02 ,  C12N9/10 ,  C12N9/12 ,  C12N9/44 ,  C12N9/64 ,  C12N9/88 ,  C12N15/85

CPC classification number: C12N9/1018 ,  A61K39/00 ,  A61K48/00 ,  C07K14/445 ,  C07K14/463 ,  C07K14/47 ,  C07K14/4712 ,  C07K14/4717 ,  C07K14/505 ,  C07K14/524 ,  C07K14/575 ,  C07K14/705 ,  C07K14/745 ,  C07K14/805 ,  C07K14/8125 ,  C12N9/0071 ,  C12N9/1051 ,  C12N9/1205 ,  C12N9/2451 ,  C12N9/644 ,  C12N9/88 ,  C12N2740/16222 ,  C12N2840/105 ,  C12N2840/60 ,  C12Y114/16001 ,  C12Y201/03003 ,  C12Y204/01025 ,  C12Y207/0104 ,  C12Y302/01033 ,  C12Y304/21022 ,  C12Y403/02001

Abstract: This invention provides a range of translatable messenger UNA (mUNA) molecules. The mUNA molecules can be translated in vitro and in vivo to provide an active polypeptide or protein, or to provide an immunization agent or vaccine component. The mUNA molecules can be used as an active agent to express an active polypeptide or protein in cells or subjects. Among other things, the mUNA molecules are useful in methods for treating rare diseases.

公开(公告)号：EP3268468A1

公开(公告)日：2018-01-17

申请号：EP16714581.2

申请日：2016-03-10

Applicant: Biopetrolia AB

Inventor： QIN, Jiufu ,  KRIVORUCHKO, Anastasia ,  DAVID, Florian ,  JIANG, Bo ,  NIELSEN, Jens

IPC: C12N9/10 ,  C12N15/52 ,  C12P13/00 ,  C12P13/10

CPC classification number: C12P13/10 ,  C12N9/0008 ,  C12N9/1018 ,  C12N9/1096 ,  C12N9/1217 ,  C12N9/78 ,  C12N9/88 ,  C12N15/52 ,  C12P13/001 ,  C12Y102/01038 ,  C12Y201/03003 ,  C12Y206/01011 ,  C12Y207/02008 ,  C12Y305/03001 ,  C12Y401/01017 ,  C12Y403/02001

Abstract: The present invention relates to the provision of genetically modified microbial cells, such as yeast cells with an improved ability for producing L-ornithine and its derivatives. Overproduction of L-ornithine is obtained in the first place by the down-regulation or attenuation of specially selected genes, wherein said genes encode enzymes involved in the L-ornithine consumption and/or degradation pathways. Further L-ornithine production ability is improved by down-regulation, attenuation, deletion or overexpression of specially selected genes, wherein said genes encode enzymes and/or proteins involved in the L-ornithine ‘acetylated derivatives cycle’, L-glutamate synthesis pathways, subcellular trafficking, TCA cycle, pyruvate carboxylation pathway, respiratory electron-transport chain, and the carbon substrates' assimilation machinery. The invention additionally provides a method to produce L-ornithine with said modified eukaryotic cells.

公开(公告)号：US11859215B2

公开(公告)日：2024-01-02

申请号：US16765604

申请日：2018-11-21

Applicant: ModernaTX, Inc.

Inventor： Zhijian Zhuo ,  Andrea Lea Frassetto ,  Paolo G. V. Martini ,  Vladimir Presnyak ,  Patrick Finn

IPC: A61P7/00 ,  C12N9/10 ,  A61K9/00 ,  A61K9/51

CPC classification number: C12N9/1018 ,  A61K9/0019 ,  A61K9/5123 ,  A61P7/00 ,  C12Y201/03003

Abstract: This disclosure relates mRNA therapy for the treatment of ornithine transcarbamylase deficiency (OTCD). mRNAs for use in the invention, when administered in vivo, encode human ornithine transcarbamylase (OTC), isoforms thereof, functional fragments thereof, and fusion proteins comprising OTC. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of OTC expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic ammonia associated with deficient OTC activity in subjects.

公开(公告)号：US20230416700A1

公开(公告)日：2023-12-28

申请号：US18339457

申请日：2023-06-22

Applicant: The Trustees of the University of Pennsylvania

Inventor： Lili Wang ,  James M. Wilson

IPC: C12N9/10 ,  C12N15/86 ,  A61K48/00

CPC classification number: C12N9/1018 ,  C12Y201/03003 ,  C12N15/86 ,  A61K48/0058 ,  C12N2750/14032 ,  C12N2750/14071 ,  C12N2750/14033 ,  C12N2710/10343 ,  C12N2740/15043 ,  C12N2750/14143 ,  C12N2800/22 ,  C12N2750/14043

Abstract: Viral vectors comprising engineered hOTC DNA and RNA sequences are provided which when delivered to a subject in need thereof are useful for treating hyperammonemia, ornithine transcarbamylase deficiency and symptoms associated therewith. Also provided are methods of using hOTC for treatment of liver fibrosis and/or cirrhosis in OTCD patients by administering hOTC.

公开(公告)号：US20180153822A1

公开(公告)日：2018-06-07

申请号：US15809680

申请日：2017-11-10

Applicant: Translate Bio, Inc.

Inventor： Shrirang Karve ,  Frank DeRosa ,  Zarna Bhavsar ,  Michael Heartlein

IPC: A61K9/51 ,  A61K38/53 ,  A61K38/44 ,  A61K38/18 ,  A61K38/17 ,  A61K38/45

CPC classification number: A61K9/5192 ,  A61K9/1272 ,  A61K9/5123 ,  A61K38/177 ,  A61K38/1816 ,  A61K38/44 ,  A61K38/45 ,  A61K38/53 ,  A61K47/22 ,  A61K47/28 ,  C12N15/88 ,  C12Y114/16001 ,  C12Y201/03003 ,  C12Y603/04005

Abstract: The present invention provides an improved process for lipid nanoparticle formulation and mRNA encapsulation. In some embodiments, the present invention provides a process of encapsulating messenger RNA (mRNA) in lipid nanoparticles comprising a step of mixing a solution of pre-formed lipid nanoparticles and mRNA.