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1. (WO2019050406) NEW THERAPY FOR POMPE DISEASE
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Pub. No.: WO/2019/050406 International Application No.: PCT/NL2018/050581
Publication Date: 14.03.2019 International Filing Date: 07.09.2018
IPC:
A61K 48/00 (2006.01) ,C12N 9/24 (2006.01) ,C12N 15/63 (2006.01)
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
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
14
Hydrolases (3.)
24
acting on glycosyl compounds (3.2)
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
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
Applicants:
ERASMUS UNIVERSITY MEDICAL CENTER ROTTERDAM [NL/NL]; Dr. Molewaterplein 50 3015 GE Rotterdam, NL
Inventors:
PIJNAPPEL, Wilhemus Wenceslaus Matthias; NL
VANDER PLOEG, Antje Tjitske; NL
VAN DER WAL, Erik; NL
KRUIJT, Jur; NL
HERRERO HERNADEZ, Pablo; NL
Agent:
JANSEN, C.M.; V.O. Carnegieplein 5 2517 KJ Den Haag, NL
Priority Data:
201951708.09.2017NL
Title (EN) NEW THERAPY FOR POMPE DISEASE
(FR) NOUVELLE THÉRAPIE POUR LA MALADIE DE POMPE
Abstract:
(EN) The invention relates to methods for gene therapy in a subject suffering from Pompe disease, comprising gene-editing of a glucosidase, acid, alpha gene (GAA) in said subject. The invention further relates to a cell culture of genetically changed, differentiated myogenic progenitor cells derived from a donor subject suffering from Pompe disease, to a vector for use in a method for gene-editing a eukaryotie cell, and to a myotube prepared from myogenic progenitor cells that have been genetically changed by gene-editing.
(FR) L'invention concerne des procédés de thérapie génique chez un sujet souffrant de la maladie de Pompe, comprenant l'édition génique d’un gène de l’alpha-glucosidase acide (GAA) chez ledit sujet. L'invention concerne en outre une culture cellulaire de cellules progénitrices myogènes différenciées génétiquement modifiées issues d'un sujet donneur souffrant de la maladie de Pompe, un vecteur destiné à être utilisé dans un procédé d'édition génique d'une cellule eucaryote, et un myotube préparé à partir de cellules progénitrices myogènes qui ont été génétiquement modifiées par édition génique.
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)