WIPO logo
Mobile | Deutsch | Español | Français | 日本語 | 한국어 | Português | Русский | 中文 | العربية |
PATENTSCOPE

Search International and National Patent Collections
World Intellectual Property Organization
Search
 
Browse
 
Translate
 
Options
 
News
 
Login
 
Help
 
Machine translation
1. (WO2017193029) CRISPR/CAS-RELATED METHODS AND COMPOSITIONS FOR TREATING DUCHENNE MUSCULAR DYSTROPHY
Latest bibliographic data on file with the International Bureau    Submit observation

Pub. No.: WO/2017/193029 International Application No.: PCT/US2017/031351
Publication Date: 09.11.2017 International Filing Date: 05.05.2017
IPC:
C07K 14/47 (2006.01) ,C12N 9/16 (2006.01) ,C12N 15/63 (2006.01) ,C12N 15/66 (2006.01) ,A61K 48/00 (2006.01)
Applicants: DUKE UNIVERSITY[US/US]; 2812 Erwin Road, Suite 306 Durham, NC 27705, US
EDITAS MEDICINE, INC.[US/US]; 11 Hurley Street Cambridge, MA 01241, US
Inventors: BUMCROT, David, A.; US
HUSTON, Nicholas, C.; US
TYCKO, Joshua, C.; US
ROBINSON-HAMM, Jacqueline; US
GERSBACH, Charles, A.; US
Agent: YEH, Sansun; US
Priority Data:
62/332,29705.05.2016US
Title (EN) CRISPR/CAS-RELATED METHODS AND COMPOSITIONS FOR TREATING DUCHENNE MUSCULAR DYSTROPHY
(FR) MÉTHODES LIÉES À CRISPR/CAS ET COMPOSITIONS DESTINÉES À TRAITER LA DYSTROPHIE MUSCULAIRE DE DUCHENNE
Abstract: front page image
(EN) Disclosed herein are vectors that targets a dystrophin gene, encoding at least one Cas9 molecule or a Cas9 fusion protein, and at least one gRNA molecule (e.g., two gRNA molecules), and compositions and cells comprising such vectors. Also provided are methods for using the vectors, compositions and cells for genome engineering (e.g., correcting a mutant dystrophin gene), and for treating DMD.
(FR) L'invention concerne des vecteurs qui ciblent un gène de la dystrophine, codant pour au moins une molécule Cas9 ou une protéine de fusion Cas9, et au moins une molécule d'ARNg (p. ex., deux molécules d'ARNg), et des compositions et des cellules contenant lesdits vecteurs. Des procédés d'utilisation desdits vecteurs, compositions et cellules pour l'ingénierie génomique (p. ex., correction d'un gène de la dystrophine mutant), et le traitement de la DMD sont en outre décrits.
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, 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)