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1. (WO2012054723) TREATMENT OF ALPHA-L-IDURONIDASE (IDUA) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPT TO IDUA
Latest bibliographic data on file with the International Bureau   

Pub. No.: WO/2012/054723 International Application No.: PCT/US2011/057097
Publication Date: 26.04.2012 International Filing Date: 20.10.2011
IPC:
C12N 15/113 (2010.01) ,C12Q 1/68 (2006.01) ,C12N 15/63 (2006.01) ,A61K 31/7088 (2006.01) ,A61K 48/00 (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
Q
MEASURING OR TESTING PROCESSES INVOLVING ENZYMES OR MICRO-ORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
1
Measuring or testing processes involving enzymes or micro-organisms; Compositions therefor; Processes of preparing such compositions
68
involving nucleic acids
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
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31
Medicinal preparations containing organic active ingredients
70
Carbohydrates; Sugars; Derivatives thereof
7088
Compounds having three or more nucleosides or nucleotides
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
Applicants:
COLLARD, Joseph [US/US]; US (UsOnly)
KHORKOVA SHERMAN, Olga [US/US]; US (UsOnly)
COITO, Carlos [PT/US]; US (UsOnly)
Gang, Shen [CN/US]; US (UsOnly)
CURNA INC. [US/US]; 4400 Biscayne Blvd. Miami, Florida 33137, US (AllExceptUS)
Inventors:
COLLARD, Joseph; US
KHORKOVA SHERMAN, Olga; US
COITO, Carlos; US
Gang, Shen; US
Agent:
BROWDER, Monte, R.; 4400 Biscayne Blvd. Miami, Florida 33137, US
Priority Data:
61/405,75822.10.2010US
Title (EN) TREATMENT OF ALPHA-L-IDURONIDASE (IDUA) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPT TO IDUA
(FR) TRAITEMENT DE MALADIES ASSOCIÉES À L'ALPHA-L-IDURONIDASE (IDUA) PAR INHIBITION DU TRANSCRIT ANTISENS ENDOGÈNE DE IDUA
Abstract:
(EN) The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of Alpha-L-Iduronidase (IDUA), in particular, by targeting natural antisense polynucleotides of Alpha-L-Iduronidase (IDUA). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of IDUA.
(FR) La présente invention concerne des oligonucléotides antisens qui modulent l'expression et/ou la fonction de l'alpha-L-iduronidase (IDUA), en particulier, par le ciblage des polynucléotides antisens endogènes de l'alpha-L-iduronidase (IDUA). L'invention concerne également l'identification de ces oligonucléotides antisens, et leur utilisation dans le traitement de maladies et de troubles associés à l'expression de IDUA.
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Designated States: AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, 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, IS, JP, KE, KG, KM, KN, KP, KR, KZ, LA, LC, LK, LR, LS, LT, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, 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, SZ, TZ, UG, ZM, ZW)
Eurasian Patent Office (AM, AZ, BY, KG, KZ, MD, 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, ML, MR, NE, SN, TD, TG)
Publication Language: English (EN)
Filing Language: English (EN)