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1. (WO2018227247) COMPOSITIONS AND METHODS FOR TREATING CONDITIONS ASSOCIATED WITH GAIN-OF-FUNCTION MUTATIONS IN KCNT1
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Pub. No.: WO/2018/227247 International Application No.: PCT/AU2018/050587
Publication Date: 20.12.2018 International Filing Date: 13.06.2018
IPC:
C12N 15/113 (2010.01) ,A61K 31/7088 (2006.01) ,A61K 31/713 (2006.01) ,A61P 25/08 (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
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
31
Medicinal preparations containing organic active ingredients
70
Carbohydrates; Sugars; Derivatives thereof
7088
Compounds having three or more nucleosides or nucleotides
713
Double-stranded nucleic acids or oligonucleotides
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
P
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
25
Drugs for disorders of the nervous system
08
Antiepileptics; Anticonvulsants
Applicants:
THE FLOREY INSTITUTE OF NEUROSCIENCE AND MENTAL HEALTH [AU/AU]; Level 5, Kenneth Myer Building 30 Royal Parade The University of Melbourne Melbourne, Victoria 3010, AU
Inventors:
PETROU, Steven; AU
Agent:
DAVIES COLLISON CAVE PTY LTD; 255 Elizabeth Street Sydney, New South Wales 2000, AU
Priority Data:
201790224213.06.2017AU
Title (EN) COMPOSITIONS AND METHODS FOR TREATING CONDITIONS ASSOCIATED WITH GAIN-OF-FUNCTION MUTATIONS IN KCNT1
(FR) COMPOSITIONS ET MÉTHODES POUR TRAITER DES ÉTATS ASSOCIÉS À DES MUTATIONS DE GAIN DE FONCTION DANS KCNT1
Abstract:
(EN) The disclosure relates generally to antisense oligonucleotides specific for KCNT1 and their use for treating diseases and conditions associated with excessive neuronal excitability and/or gain-of-function mutations of KCNT1.
(FR) L'invention concerne d'une manière générale des oligonucléotides antisens spécifiques pour KCNT1 et leur utilisation pour le traitement de maladies et d'états associés à une excitabilité neuronale excessive et/ou à des mutations de gain de fonction de KCNT1.
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: English (EN)
Filing Language: English (EN)