(12) International Application Status Report

Received at International Bureau: 11 October 2018 (11.10.2018)

Information valid as of: 28 May 2019 (28.05.2019)

Report generated on: 24 August 2019 (24.08.2019)

(10) Publication number: (43) Publication date: (26) Publication language:
WO 2019/06649004 April 2019 (04.04.2019) Korean (KO)

(21) Application number: (22) Filing date: (25) Filing language:
PCT/KR2018/01142427 September 2018 (27.09.2018) Korean (KO)

(31) Priority number(s): (32) Priority date(s): (33) Priority status:
62/564,478 (US)28 September 2017 (28.09.2017) Priority document received (in compliance with PCT Rule 17.1)
62/565,868 (US)29 September 2017 (29.09.2017) Priority document received (in compliance with PCT Rule 17.1)

(51) International Patent Classification:
C12N 15/113 (2010.01); C12N 9/22 (2006.01); C12N 15/10 (2006.01); C12N 15/86 (2006.01); C12N 15/90 (2006.01); A61K 48/00 (2006.01)

(71) Applicant(s):
TOOLGEN INCORPORATED [KR/KR]; #1204~1206, 219, Gasan digital 1-ro Geumcheon-gu Seoul 08501 (KR) (for all designated states)

(72) Inventor(s):
KIM, Seok Joong; #501-1802, 30, Seocho-daero 74-gil Seocho-gu Seoul 06622 (KR)
SONG, Dong Woo; 60-4, Siheung-daero 145-gil Geumcheon-gu Seoul 08532 (KR)
LEE, Jae Young; #101-602, 81, Hyoryeong-ro 68-gil Seocho-gu Seoul 06721 (KR)
LEE, Jung Min; #5-603, 20, Gukjegeumyung-ro 7-gil Yeongdeungpo-gu Seoul 07340 (KR)
CHO, Gyu Bon; #403, 42, Yeouidaebang-ro, 22ka-gil Dongjak-gu Seoul 07059 (KR)
BAE, Hee Sook; #5-801, 12, Gogang-ro 40beonna-gil Bucheon-si Gyeonggi-do 14413 (KR)

(74) Agent(s):
IPS PATENT FIRM; 5th Fl., 14, Banpo-daero 23-gil Seocho-gu Seoul 06656 (KR)

(54) Title (EN): ARTIFICIAL GENOME MANIPULATION FOR GENE EXPRESSION REGULATION
(54) Title (FR): MANIPULATION ARTIFICIELLE DE GÉNOME POUR LA RÉGULATION D'EXPRESSION GÉNIQUE
(54) Title (KO): 유전자 발현 조절을 위한 인위적인 게놈 조작

(57) Abstract:
(EN): The present invention relates to an expression control composition for regulating the expression of repeated genes or a method using the same. More specifically, the present invention relates to a composition for expression regulation, comprising a guide nucleic acid capable of targeting a transcription regulatory region of repeated genes, and a method for regulating the expression of repeated genes, using the composition to artificially manipulate and/or modify a transcription regulatory region of the repeated genes. In addition, the present invention relates to a method for treating or alleviating a disease caused by gene repetition, using an expression control composition for regulating the expression of repeated genes.
(FR): La présente invention concerne une composition de contrôle d'expression pour réguler l'expression de gènes répétés ou un procédé l'utilisant. Plus spécifiquement, la présente invention concerne une composition pour la régulation d'expression, comprenant un acide nucléique de guidage capable de cibler une région régulatrice de transcription de gènes répétés, et un procédé de régulation de l'expression de gènes répétés, utilisant la composition pour manipuler et/ou modifier artificiellement une région régulatrice de transcription des gènes répétés. De plus, la présente invention concerne une méthode pour traiter ou soulager une maladie provoquée par la répétition de gènes, à l'aide d'une composition de contrôle d'expression pour réguler l'expression de gènes répétés.
(KO): 본 발명은 중복 유전자의 발현을 조절하기 위한 발현 조절용 조성물 또는 이를 이용한 방법에 관한 것이다. 보다 구체적으로, 중복 유전자의 전사 조절 영역을 표적화할 수 있는 가이드핵산을 포함하는 발현 조절용 조성물 및 이를 이용한 중복 유전자의 전사 조절 영역을 인위적으로 조작 및/또는 변형시켜 중복 유전자의 발현을 조절하는 방법에 관한 것이다. 또한 중복 유전자의 발현을 조절하기 위한 발현 조절용 조성물을 이용하여 유전자 중복에 의해 발생하는 질환을 치료 또는 개선하는 방법에 관한 것이다.

International search report:
Received at International Bureau: 09 May 2019 (09.05.2019) [KR]

International Report on Patentability (IPRP) Chapter II of the PCT:
Not available

(81) 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
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 (OAPI) : BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG
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 (EAPO) : AM, AZ, BY, KG, KZ, RU, TJ, TM