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1. (WO2018226785) SELF-REGULATING AAV VECTORS FOR SAFE EXPRESSION OF MECP2 IN RETT SYNDROME
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Pub. No.: WO/2018/226785 International Application No.: PCT/US2018/036200
Publication Date: 13.12.2018 International Filing Date: 06.06.2018
IPC:
C12N 15/86 (2006.01) ,C12N 15/117 (2010.01)
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
79
Vectors or expression systems specially adapted for eukaryotic hosts
85
for animal cells
86
Viral vectors
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
117
Nucleic acids having immunomodulatory properties, e.g. containing CpG-motifs
Applicants:
UNIVERSITY OF MASSACHUSETTS [US/US]; One Beacon Street 31st Floor Boston, MA 02108, US
Inventors:
ESTEVES, Miguel, Sena; US
GAO, Guangping; US
GREEN, Michael, R.; US
WANG, Dan; US
SIMONE, Tessa, Mercedes; US
Agent:
MACDONALD, Kevin; US
Priority Data:
62/516,06006.06.2017US
Title (EN) SELF-REGULATING AAV VECTORS FOR SAFE EXPRESSION OF MECP2 IN RETT SYNDROME
(FR) VECTEURS AAV À AUTORÉGULATION POUR L'EXPRESSION SANS RISQUE DE MECP2 DANS LE SYNDROME DE RETT
Abstract:
(EN) In some aspects, the disclosure relates to compositions and methods of engineering a transgene. In some embodiments, the disclosure provides self -regulating recombinant nucleic acids, viral vectors and pharmaceutical compositions comprising a MeCP2 transgene. In some embodiments, compositions and methods described by the disclosure are useful for treating diseases and disorders associated with a loss of function mutation, for example Rett syndrome.
(FR) Selon certains aspects, cette divulgation concerne des compositions et des méthodes d'ingénierie d'un transgène. Dans certains modes de réalisation, la divulgation concerne des acides nucléiques recombinés à auto-régulation, des vecteurs viraux et des compositions pharmaceutiques comprenant un transgène MeCP2. Dans d'autres, les compositions et les procédés ci-décrits sont utiles pour traiter des maladies et des troubles associés à une perte de mutation fonctionnelle, par exemple le syndrome de Rett.
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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)