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1. (WO2016179112) PRECISE DELETION OF CHROMOSCOMAL SEQUENCES IN VIVO AND TREATMENT OF NUCLEOTIDE REPEAT EXPANSION DISORDERS USING ENGINEERED NUCLEASES
Latest bibliographic data on file with the International Bureau   

Pub. No.:    WO/2016/179112    International Application No.:    PCT/US2016/030460
Publication Date: 10.11.2016 International Filing Date: 02.05.2016
IPC:
C12N 9/16 (2006.01), C12N 15/11 (2006.01)
Applicants: PRECISION BIOSCIENCES, INC. [US/US]; 302 East Pettigrew Street Dibrell Building, Suite A-100 Durham, NC 27701 (US).
SMITH, James, Jefferson [US/US]; (US).
BARTSEVICH, Victor [US/US]; (US)
Inventors: SMITH, James, Jefferson; (US).
BARTSEVICH, Victor; (US).
JANTZ, Derek; (US)
Agent: TWOMEY, Michael, J.; (US)
Priority Data:
62/155,838 01.05.2015 US
Title (EN) PRECISE DELETION OF CHROMOSCOMAL SEQUENCES IN VIVO AND TREATMENT OF NUCLEOTIDE REPEAT EXPANSION DISORDERS USING ENGINEERED NUCLEASES
(FR) SUPPRESSION PRÉCISE DE SÉQUENCES CHROMOSOMIQUES IN VIVO ET TRAITEMENT DE TROUBLES À EXPANSION DE NUCLÉOTIDES RÉPÉTÉS À L'AIDE D'ENZYMES DE RESTRICTION
Abstract: front page image
(EN)The present invention provides a method of treating a nucleotide repeat expansion disorder comprising delivering a pair of engineered nucleases, or genes encoding engineered nucleases, to the cells of a patient such that the two nucleases excise the nucleotide repeat responsible for the disease permanently from the genome. The invention provides a general method for treating nucleotide repeat expansion disorders and engineered nucleases suitable for practicing the method. The invention further provides vectors and techniques for delivering engineered nucleases to patient cells.
(FR)La présente invention concerne un procédé de traitement d'un trouble à expansion de nucléotides répétés consistant à administrer une paire d'enzymes de restriction, ou des gènes codant pour des enzymes de restriction, les cellules d'un patient de telle sorte que les deux nucléases excisent du génome le nucléotide répété à l'origine de la maladie de manière définitive. L'invention concerne un procédé général permettant de traiter des troubles à expansion de nucléotides répétés et des enzymes de restriction appropriées pour mettre en ouvre le procédé. L'invention concerne en outre des vecteurs et des techniques d'administration d'enzymes de restriction à des cellules du patient.
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 (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)