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1. (WO2018002762) MATERIALS AND METHODS FOR TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) AND OTHER RELATED DISORDERS
Latest bibliographic data on file with the International Bureau    Submit observation

Pub. No.:    WO/2018/002762    International Application No.:    PCT/IB2017/053575
Publication Date: 04.01.2018 International Filing Date: 15.06.2017
IPC:
A61K 38/44 (2006.01), C12N 15/90 (2006.01), C12N 9/22 (2006.01)
Applicants: CRISPR THERAPEUTICS AG [CH/CH]; Baarerstrasse 14 6300 Zug (CH)
Inventors: LUNDBERG, Ante Sven; (US).
KULKARNI, Samarth; (US).
KLEIN, Lawrence; (US).
PADMANABHAN, Hari Kumar; (US)
Priority Data:
62/355,921 29.06.2016 US
62/461,862 22.02.2017 US
Title (EN) MATERIALS AND METHODS FOR TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) AND OTHER RELATED DISORDERS
(FR) MATÉRIAUX ET MÉTHODES POUR TRAITER LA SCLÉROSE LATÉRALE AMYOTROPHIQUE (ALS) ET D'AUTRES TROUBLES ASSOCIÉS
Abstract: front page image
(EN)The present disclosure provides materials and methods for treating a patient with one or more conditions associated with SOD1 whether ex vivo or in vivo. In addition, the present disclosure provides materials and methods for editing and/or modulating the expression of SOD1 gene in a cell by genome editing.
(FR)La présente invention concerne des matériaux et des méthodes pour traiter un patient présentant une ou plusieurs maladies associées à SOD1 ex vivo ou in vivo. La présente invention concerne également des matériaux et des méthodes qui permettent d'éditer et/ou de moduler l’expression du gène SOD1 dans une cellule par édition du génome.
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 (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW)
Eurasian Patent Organization (AM, AZ, BY, KG, KZ, RU, TJ, TM)
European Patent Office (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)