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1. (WO2017035659) METHOD AND COMPOSITIONS FOR REMOVING DUPLICATED COPY NUMBER VARIATIONS (CNVS) FOR GENETIC DISORDERS AND RELATED USES
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Pub. No.: WO/2017/035659 International Application No.: PCT/CA2016/051041
Publication Date: 09.03.2017 International Filing Date: 01.09.2016
IPC:
A61K 38/46 (2006.01) ,A01K 67/027 (2006.01) ,A61K 31/7088 (2006.01) ,A61K 48/00 (2006.01) ,A61P 21/00 (2006.01) ,C07H 21/00 (2006.01) ,C12N 15/00 (2006.01) ,C12N 15/09 (2006.01) ,C12N 15/11 (2006.01) ,C12Q 1/00 (2006.01) ,C12Q 1/68 (2006.01)
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
46
Hydrolases (3)
A HUMAN NECESSITIES
01
AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
K
ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
67
Rearing or breeding animals, not otherwise provided for; New breeds of animals
027
New breeds of vertebrates
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
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
P
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
21
Drugs for disorders of the muscular or neuromuscular system
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
H
SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
21
Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. 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
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
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
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
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
Applicants:
THE HOSPITAL FOR SICK CHILDREN [CA/CA]; 555 University Avenue Toronto, Ontario M5G 1X8, CA
Inventors:
COHN, Ronald; CA
WOJTAL, Daria; CA
KEMALADEWI, Dwi; CA
IVAKINE, Zhenya; CA
Agent:
NADOR, Anita; CA
TISDALL, Grant; CA
Priority Data:
62/212,93401.09.2015US
Title (EN) METHOD AND COMPOSITIONS FOR REMOVING DUPLICATED COPY NUMBER VARIATIONS (CNVS) FOR GENETIC DISORDERS AND RELATED USES
(FR) PROCÉDÉ ET COMPOSITIONS PERMETTANT D'ÉLIMINER DES VARIATIONS DE NOMBRE DE COPIES (CNV) DUPLIQUÉES POUR DES TROUBLES GÉNÉTIQUES ET UTILISATIONS ASSOCIÉES
Abstract:
(EN) In one aspect, the Invention relates to methods and compositions to remove replicate genetic material, such as duplicate copy number variations (CNVs) present in genetic material using targeted endonuclease technology with one guide, such as one single guide RNA. In another aspect, the invention relates to methods and compositions useful for removing replicate or duplicate genetic material. In another aspect the invention is useful for the treatment and prevention of conditions that are caused by replicate genetic material, including duplicate CNVs, such as a certain types of Duchenne Muscular Dystrophy and or MECP2 duplication syndrome. In another aspect the invention provides novel genetically engineered animals with duplicate CNVs useful to screen potential therapies.
(FR) Dans un aspect, l'invention concerne des procédés et des compositions permettant d'éliminer du matériel génétique répliqué, tel que des variations de nombre de copies (CNV) dupliquées présentes dans un matériel génétique au moyen d'une technologie d'endonucléase ciblée avec un guide, tel qu'un ARN guide unique. Dans un autre aspect, l'invention concerne des procédés et des compositions utiles pour éliminer un matériel génétique répliqué ou dupliqué. Dans un autre aspect, l'invention est utile pour le traitement et la prévention d'affections qui sont provoquées par un matériel génétique répliqué, notamment des CNV dupliqués, comme certains types de dystrophie musculaire de Duchenne et/ou de syndrome de duplication de MECP2. Dans un autre aspect, l'invention concerne de nouveaux animaux génétiquement modifiés avec des CNV dupliqués utiles pour cribler des thérapies potentielles.
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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, 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)