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1. (WO2016063264) PERMANENT EPIGENETIC GENE SILENCING
Latest bibliographic data on file with the International Bureau   

Pub. No.: WO/2016/063264 International Application No.: PCT/IB2015/058202
Publication Date: 28.04.2016 International Filing Date: 23.10.2015
IPC:
C12N 15/85 (2006.01) ,A61K 48/00 (2006.01) ,A61K 38/45 (2006.01) ,C12N 9/10 (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
63
Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
79
Vectors or expression systems specially adapted for eukaryotic hosts
85
for animal cells
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48
Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
38
Medicinal preparations containing peptides
16
Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
43
Enzymes; Proenzymes; Derivatives thereof
45
Transferases (2)
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
10
Transferases (2.)
Applicants: OSPEDALE SAN RAFFAELE S.R.L.[IT/IT]; Via Olgettina, 60 I-20132 Milan, IT
FONDAZIONE TELETHON[IT/IT]; Via Varese 16B I-00185 Rome, IT
Inventors: NALDINI, Luigi; IT
LOMBARDO, Angelo Leone; IT
AMABILE, Angelo; IT
MIGLIARA, Alessandro; IT
Agent: O'BRIEN, Simon; GB
Priority Data:
1418965.824.10.2014GB
Title (EN) PERMANENT EPIGENETIC GENE SILENCING
(FR) SILENÇAGE GÉNIQUE ÉPIGÉNÉTIQUE PERMANENT
Abstract:
(EN) A product comprising two or more artificial transcription repressors (ATRs), or polynucleotides encoding therefor, selected from groups (a), (b), (c) or (d): (a) an ATR comprising a DNA-binding domain operably linked to a KRAB domain or homologue thereof; (b) an ATR comprising a DNA-binding domain operably linked to a DNMT3A, DNMT3B or DNMT1 domain or homologue thereof; (c) an ATR comprising a DNA-binding domain operably linked to a DNMT3L domain or homologue thereof; and (d) an ATR comprising a DNA-binding domain operably linked to a SETDB1 domain or homologue thereof, wherein at least two of the ATRs are selected from different groups (a), (b), (c) or (d).
(FR) La présente invention concerne un produit contenant au moins deux répresseurs de transcription artificiels (ATR), ou des polynucléotides les codant, choisis dans les groupes (a), (b), (c) ou (d) : (a) un ATR comprenant un domaine de liaison à l'ADN lié de manière fonctionnelle à un domaine KRAB ou son homologue; (b) un ATR comprenant un domaine de liaison à l'ADN lié de manière fonctionnelle à un domaine DNMT3A, DNMT3B ou DNMT1 ou son homologue; (c) un ATR comprenant un domaine de liaison à l'ADN lié de manière fonctionnelle à un domaine DNMT3L ou son homologue; et (d) un ATR comprenant un domaine de liaison à l'ADN lié de manière fonctionnelle à un domaine SETDB1 ou son homologue. Au moins deux des ATR sont choisis dans les différents groupes (a), (b), (c) ou (d).
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, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KN, KP, KR, 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: English (EN)
Filing Language: English (EN)