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1. (WO2019046341) METHODS AND COMPOSITIONS FOR TREATING CONE-ROD RETINAL DYSTROPHY
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Pub. No.: WO/2019/046341 International Application No.: PCT/US2018/048405
Publication Date: 07.03.2019 International Filing Date: 28.08.2018
IPC:
A61K 48/00 (2006.01) ,C12N 15/85 (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
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
79
Vectors or expression systems specially adapted for eukaryotic hosts
85
for animal cells
Applicants:
UNIVERSITY OF FLORIDA RESEARCH FOUNDATION, INCORPORATED [US/US]; 223 Grinter Hall Gainesville, FL 32611, US
EDITAS MEDICINE, INC. [US/US]; 11 Hurley St Cambridge, MA 02141, US
Inventors:
BOYE, Shannon, E.; US
BOYE, Sanford, L.; US
MAEDER, Morgan; US
Agent:
WALLER, Patrick, R.H.; US
Priority Data:
62/551,21228.08.2017US
62/664,06327.04.2018US
Title (EN) METHODS AND COMPOSITIONS FOR TREATING CONE-ROD RETINAL DYSTROPHY
(FR) MÉTHODES ET COMPOSITIONS POUR TRAITER UNE DYSTROPHIE RÉTINIENNE DES CÔNES ET DES BÂTONNETS
Abstract:
(EN) Provided herein are methods and compositions for treating an eye disorder, for example CORD6. Aspects of the disclosure relate to knocking out an autosomal dominant mutant GUCY2D gene.
(FR) L'invention concerne des méthodes et des compositions pour traiter un trouble oculaire, par exemple CORD6. Des aspects de l'invention concernent le knock-out d'un gène GUCY2D mutant dominant autosomique.
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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)