Some content of this application is unavailable at the moment.
If this situation persist, please contact us atFeedback&Contact
1. (WO2019062522) SGRNA, ENGINEERED CAS9 PROTEIN, AND KIT
Latest bibliographic data on file with the International Bureau    Submit observation

Pub. No.: WO/2019/062522 International Application No.: PCT/CN2018/104650
Publication Date: 04.04.2019 International Filing Date: 07.09.2018
IPC:
C12N 15/113 (2010.01) ,C12N 15/90 (2006.01) ,C12N 9/22 (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
87
Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
90
Stable introduction of foreign DNA into chromosome
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
9
Enzymes, e.g. ligases (6.); Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating, or purifying enzymes
14
Hydrolases (3.)
16
acting on ester bonds (3.1)
22
Ribonucleases
Applicants:
深圳三智医学科技有限公司 SHENZHEN CHANGENE MEDICAL TECHNOLOGY CO., LTD. [CN/CN]; 中国广东省深圳市 南山区西丽街道茶光路1089号深圳集成电路设计应用产业园207-2 207-2# Shenzhen IC Design & Application Industrial Park, 1089 Chaguang Road, Nanshan District Shenzhen, Guangdong 518000, CN
Inventors:
张芸 ZHANG, Yun; CN
姜舒 JIANG, Shu; CN
纪惜銮 JI, Xiluan; CN
刘婕 LIU, Jie; CN
郭明 GUO, Ming; CN
罗朝霞 LUO, Zhaoxia; CN
杨顺 YANG, Shun; CN
Agent:
深圳市博锐专利事务所 BORSAM INTELLECTUAL PROPERTY; 中国广东省深圳市 福田区梅林街道福田国际电子商务产业园科技楼1002 Room 1002, Keji Building, Futian International E-Commerce Indu Meilin Street, Futian District Shenzhen, Guangdong 518049, CN
Priority Data:
201710922540.930.09.2017CN
Title (EN) SGRNA, ENGINEERED CAS9 PROTEIN, AND KIT
(FR) ARNSG, PROTÉINE CAS9 MODIFIÉE, ET KIT
(ZH) 一种sgRNA、改造的Cas9蛋白及试剂盒
Abstract:
(EN) The present invention provides an sgRNA, an engineered Cas9 protein, and a kit. The kit comprises: an sgRNA specifically targeting an HBB gene or a vector comprising the sgRNA nucleotide sequence; and/or an engineered Cas9 enzyme or a sequence encoding the engineered Cas9 enzyme or a vector comprising the sequence encoding the engineered Cas9 enzyme; and/or a donor gene sequence for repairing a β-thalassemia HBB gene or a vector comprising the donor gene sequence. The application further provides an application of the kit for cleavage or repair of an HBB gene, or more specifically for the repair of an HBB gene of autologous hematopoietic stem cells in β-thalassemia patients.
(FR) La présente invention concerne un ARNsg, une protéine Cas9 modifiée, et un kit. Le kit comprend : un ARNsg ciblant spécifiquement un gène HBB ou un vecteur comprenant la séquence nucléotidique d'ARNsg; et/ou une enzyme Cas9 modifiée ou une séquence codant pour l'enzyme Cas9 modifiée ou un vecteur comprenant la séquence codant pour l'enzyme Cas9 modifiée; et/ou une séquence de gène donneur pour réparer un gène HBB de β-thalassémie ou un vecteur comprenant la séquence de gène donneur. L'application concerne en outre un applicateur du kit de clivage ou de réparation d'un gène HBB, ou plus spécifiquement pour la réparation d'un gène HBB de cellules souches hématopoïétiques autologues chez des patients présentant une β-thalassémie.
(ZH) 提供了一种sgRNA、改造的Cas9蛋白及试剂盒。该试剂盒包括:特异性靶向HBB基因的sgRNA或包含该sgRNA核苷酸序列的载体;和/或改造的Cas9酶或编码该改造的Cas9酶的序列或包含编码该改造的Cas9酶序列的载体;和/或用于修复β-地中海贫血HBB基因的供体基因序列或包含所述供体基因序列的载体。还提供了所述试剂盒在切割或修复HBB基因中的应用,尤其是在修复β-地中海贫血患者自体造血干细胞的HBB基因中的应用。
front page image
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, JO, 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)