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1. (WO2017035342) DRUG DISCOVERY PLATFORM FOR DUCHENNE CARDIOMYOPATHY
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Pub. No.: WO/2017/035342 International Application No.: PCT/US2016/048671
Publication Date: 02.03.2017 International Filing Date: 25.08.2016
IPC:
C12N 15/85 (2006.01) ,C12Q 1/00 (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
63
Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
79
Vectors or expression systems specially adapted for eukaryotic hosts
85
for animal cells
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
Applicants:
UNIVERSITY OF WASHINGTON [US/US]; 4545 Roosevelt Way NE, Suite 400 Seattle, Washington 98105, US
Inventors:
CHILDERS, Martin K.; US
Agent:
OH, Zhi-Xiang (Alex); US
Priority Data:
62/210,68327.08.2015US
Title (EN) DRUG DISCOVERY PLATFORM FOR DUCHENNE CARDIOMYOPATHY
(FR) PLATE-FORME DE DÉCOUVERTE DE MÉDICAMENT POUR LA CARDIOMYOPATHIE DE DUCHENNE
Abstract:
(EN) Methods of screening agents in a cardiomyocyte population are provided. The cardiomyocyte population may be differentiated from a dystrophin knockout iPSC line. High-throughput methods of screening agents in a cardiomyocyte population that has been differentiated from a dystrophin knockout iPSC line are also provided. The methods may include determining an effect of the agents on membrane barrier function by using a cell viability assay. Methods of making dystrophin knockout iPSC lines, making dystrophin knockout iPSC derived cardiomyocytes, and modeling dystrophin deficient cardiomyopathy are also provided.
(FR) L'invention concerne des procédés de criblage d'agents dans une population de cardiomyocytes. La population de cardiomyocytes peut s'être différenciée à partir d'une lignée de CSPI à dystrophine inactivée. L'invention concerne également des procédés de criblage à haut débit d'agents dans une population de cardiomyocytes qui s'est différenciée à partir d'une lignée de CSPI à dystrophine inactivée. Les procédés peuvent comprendre la détermination d'un effet des agents sur la fonction de barrière membranaire à l'aide d'un test de viabilité cellulaire. L'invention concerne également des procédés de production cardiomyocytes dérivés de CSPI à dystrophine inactivée, et de modélisation d'une cardiomyopathie déficiente en dystrophine.
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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)