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1. (WO2018080541) METHOD FOR TREATING AUTOIMMUNE DISEASE USING CD4 T-CELLS WITH ENGINEERED STABILIZATION OF EXPRESSION OF ENDOGENNOUS FOXP3 GENE
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Pub. No.: WO/2018/080541 International Application No.: PCT/US2016/059729
Publication Date: 03.05.2018 International Filing Date: 31.10.2016
IPC:
A61K 48/00 (2006.01) ,C12N 5/0783 (2010.01) ,C12N 15/66 (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
5
Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
07
Animal cells or tissues
071
Vertebrate cells or tissues, e.g. human cells or tissues
078
Cells from blood or from the immune system
0783
T cells; NK cells; Progenitors of T or NK cells
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
66
General methods for inserting a gene into a vector to form a recombinant vector using cleavage and ligation; Use of non-functional linkers or adaptors, e.g. linkers containing the sequence for a restriction endonuclease
Applicants:
SEATTLE CHILDREN'S HOSPITAL (DBA SEATTLE CHILDREN'S RESEARCH INSTITUTE) [US/US]; 1900 Ninth Avenue Seattle, WA 98101, US
Inventors:
SCHARENBERG, Andrew M.; US
RAWLINGS, David J.; US
TORGERSON, Troy; US
Agent:
FURMAN, Eric S.; US
Priority Data:
Title (EN) METHOD FOR TREATING AUTOIMMUNE DISEASE USING CD4 T-CELLS WITH ENGINEERED STABILIZATION OF EXPRESSION OF ENDOGENNOUS FOXP3 GENE
(FR) MÉTHODE DE TRAITEMENT DE MALADIES AUTO-IMMUNES À L'AIDE DE CELLULES T CD4 MODIFIÉES EN VUE D'OBTENIR UNE STABILISATION DE L'EXPRESSION DU GÈNE FOXP3 ENDOGÈNE
Abstract:
(EN) Disclosed are methods of making a genetically cell that expressed FOXP3 and methods of treatment. In some embodiments, the method can providing a first nucleotide sequence, wherein the first nucleotide sequence comprises a coding strand, the coding strand comprising one or more regulatory elements and a FOXP3 gene or portion thereof providing a nuclease and performing a gene editing process on the first nucleotide sequence, which edits said one or more regulatory elements, and optionally edits the FOXP3 gene or portion thereof. Methods of treating a subject suffering from an autoimmune disease and subjects suffering the effects of organ transplantation are also provided.
(FR) L'invention concerne des méthodes permettant de produire des cellules génétiquement modifiées qui expriment FOXP3, et des méthodes de traitement. Dans certains modes de réalisation, la méthode peut fournir une première séquence nucléotidique, la première séquence nucléotidique comprenant un brin codant, le brin codant comprenant un ou plusieurs éléments régulateurs et le gène FOXP3 ou une partie de ce dernier fournissant une nucléase, et réaliser un processus d'édition de gène sur la première séquence nucléotidique, qui édite ledit ou lesdits éléments régulateurs, et de façon optionnelle édite le gène FOXP3 ou une partie de ce dernier. L'invention concerne également des méthodes de traitement de patients souffrant de maladie auto-immune et de patients souffrant des effets d'une greffe d'organe.
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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, 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)