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1. (WO2018200635) METHODS OF TREATING RHEUMATOID ARTHRITIS USING RNA-GUIDED GENOME EDITING OF HLA GENE
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Pub. No.: WO/2018/200635 International Application No.: PCT/US2018/029302
Publication Date: 01.11.2018 International Filing Date: 25.04.2018
IPC:
C12N 15/63 (2006.01) ,C12N 15/11 (2006.01) ,C12N 15/90 (2006.01) ,A61K 35/14 (2015.01)
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
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
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
35
Medicinal preparations containing material or reaction products thereof with undetermined constitution
12
Materials from mammals or birds
14
Blood
Applicants:
FREED, Brian [US/US]; US (US)
ANDERSON, Kirsten [US/US]; US (US)
ROARK, Christina [US/US]; US (US)
MATSUDA, Jennifer [US/US]; US (US)
THE REGENTS OF THE UNIVERSITY OF COLORADO, A BODY CORPORATE [US/US]; 1800 Grant Street, 8th Floor Denver, Colorado 80203, US
Inventors:
FREED, Brian; US
ANDERSON, Kirsten; US
ROARK, Christina; US
MATSUDA, Jennifer; US
Agent:
TRAVER, Robert, D.; US
Priority Data:
62/491,48728.04.2017US
Title (EN) METHODS OF TREATING RHEUMATOID ARTHRITIS USING RNA-GUIDED GENOME EDITING OF HLA GENE
(FR) MÉTHODES DE TRAITEMENT DE LA POLYARTHRITE RHUMATOÏDE PAR ÉDITION GÉNOMIQUE GUIDÉE PAR L'ARN D'UN GÈNE HLA
Abstract:
(EN) Methods of preventing or treating rheumatoid arthritis (RA) in a subject by introducing the DRB1*04:01K71E mutation that is resistant to RA. The resistant allele is introduced into the subject having or at risk of developing RA, using a HLA CRISPR/Cas9 vector that targets codon 71 in the HLA allele DRB1*04:01, introducing a single A to G point mutation in codon 71 by homology directed repair to alter the lysine at position 71 of the expressed protein to glutamic acid. This modified allele is affected in the subject's hematopoietic stem cells, which are then expanded and transplanted back into the patient. This microgene therapy confers RA-resistance via an autologous transplant. The invention includes isolated nucleic acids, vectors, recombinant viruses, cells, and pharmaceutical compositions to modify the HLA DRB1*04:01 allele.
(FR) L'invention concerne des méthodes de prévention ou de traitement de la polyarthrite rhumatoïde (RA) chez un sujet par introduction de la mutation DRB1*04:01K71E qui est résistante à la RA. L'allèle résistant est introduit chez le sujet ayant ou à risque de développer une RA, à l'aide d'un vecteur HLA CRISPR/Cas9 qui cible le codon 71 dans l'allèle HLA DRB1*04:01, et introduit une mutation ponctuelle A à G unique dans le codon 71 par une réparation dirigée par homologie destinée à modifier la lysine à la position 71 de la protéine exprimée en acide glutamique. Cet allèle modifié est affecté dans les cellules souches hématopoïétiques du sujet, qui sont ensuite expansées et regreffées chez le patient. Cette thérapie microgénique confère une résistance à la RA par le biais d'une greffe autologue. Des acides nucléiques isolés, des vecteurs, des virus recombinés, des cellules et des compositions pharmaceutiques permettant de modifier l'allèle HLA DRB1*04:01 sont en outre décrits.
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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, 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)