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1. (WO2018179578) METHOD FOR INDUCING EXON SKIPPING BY GENOME EDITING
Latest bibliographic data on file with the International BureauSubmit observation

Pub. No.: WO/2018/179578 International Application No.: PCT/JP2017/041756
Publication Date: 04.10.2018 International Filing Date: 21.11.2017
IPC:
C12N 15/09 (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
Applicants:
国立大学法人京都大学 KYOTO UNIVERSITY [JP/JP]; 京都府京都市左京区吉田本町36番地1 36-1, Yoshida-honmachi, Sakyo-ku, Kyoto-shi, Kyoto 6068501, JP
Inventors:
李 紅梅 LI, Hongmei; JP
笹川 典子 SASAKAWA, Noriko; JP
堀田 秋津 HOTTA, Akitsu; JP
Agent:
佐貫 伸一 SANUKI, Shinichi; JP
丹羽 武司 NIWA, Takeshi; JP
Priority Data:
2017-06890930.03.2017JP
Title (EN) METHOD FOR INDUCING EXON SKIPPING BY GENOME EDITING
(FR) PROCÉDÉ POUR INDUIRE UN SAUT D'EXON PAR ÉDITION GÉNOMIQUE
(JA) ゲノム編集によるエクソンスキッピング誘導方法
Abstract:
(EN) A method for skipping a target exon of a desired gene in a genome using CRISPR-Cas and a guide RNA, characterized in that the guide RNA has a spacer sequence wherein the CRISPR-Cas cleavage site is positioned within 80 bases apart from a splice donor site immediately before the target exon or a splice acceptor site immediately after the target exon.
(FR) L'invention concerne un procédé pour sauter un exon cible d'un gène souhaité dans un génome à l'aide de CRISPR-Cas et d'un ARN guide, caractérisé en ce que l'ARN guide présente une séquence d'espacement, le site de clivage par CRISPR-Cas étant positionné dans les 80 bases à partir d'un site donneur d'épissage immédiatement en amont de l'exon cible ou d'un site accepteur d'épissage immédiatement en aval de l'exon cible.
(JA) CRISPR-CasおよびガイドRNAを使用したゲノム上の目的遺伝子の標的エクソンをスキップするための方法であって、前記ガイドRNA は、CRISPR-Casによる切断部位が標的エクソンの直前のスプライスドナー部位または標的エクソンの直後のスプライスアクセプター部位から80塩基以内に配置されるようなスペーサー配列を有することを特徴とする方法。
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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 Organization (AM, AZ, BY, KG, KZ, RU, TJ, TM)
European Patent Office (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: Japanese (JA)
Filing Language: Japanese (JA)