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1. (WO2017109757) MATERIALS AND METHODS FOR TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS AND/OR FRONTAL TEMPORAL LOBULAR DEGENERATION
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Pub. No.: WO/2017/109757 International Application No.: PCT/IB2016/057958
Publication Date: 29.06.2017 International Filing Date: 22.12.2016
IPC:
C12N 15/113 (2010.01) ,C12N 15/90 (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
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
87
Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
90
Stable introduction of foreign DNA into chromosome
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:
CRISPR THERAPEUTICS AG [CH/CH]; Baarerstrasse 14 Zug V8 CH-6300, CH
Inventors:
COWAN, Chad Albert; US
KABADI, Ami Meda; US
LUNDBERG, Ante Sven; US
Priority Data:
62/324,28118.04.2016US
62/387,42423.12.2015US
Title (EN) MATERIALS AND METHODS FOR TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS AND/OR FRONTAL TEMPORAL LOBULAR DEGENERATION
(FR) MATÉRIAUX ET PROCÉDÉS DE TRAITEMENT DE LA SCLÉROSE LATÉRALE AMYOTROPHIQUE ET/OU DE LA DÉGÉNÉRESCENCE LOBAIRE FRONTOTEMPORALE
Abstract:
(EN) The present application provides materials and methods for treating a patient with Amyotrophic Lateral Sclerosis (ALS) and/or Frontaltemporal Lobular Degeneration (FTLD), both ex vivo and in vivo. In addition, the present application provides materials and methods for editing to modulate the expression, function or activity of the C9ORF72 gene in a cell by genome editing.
(FR) La présente invention concerne des matériaux et des procédés permettant de traiter un patient souffrant de sclérose latérale amyotrophique (SLA) et/ou de dégénérescence lobaire frontotemporale (DLFT), ex vivo et in vivo. En outre, la présente invention concerne des matériaux et des procédés pour l'édition visant à moduler l'expression, la fonction ou l'activité du gène C9ORF72 dans une cellule par édition génique.
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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, DJ, 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, 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)