Some content of this application is unavailable at the moment.
If this situation persist, please contact us atFeedback&Contact
1. (WO2017151884) THERAPY FOR FRONTOTEMPORAL DEMENTIA
Latest bibliographic data on file with the International Bureau    Submit observation

Pub. No.: WO/2017/151884 International Application No.: PCT/US2017/020397
Publication Date: 08.09.2017 International Filing Date: 02.03.2017
IPC:
A61K 48/00 (2006.01) ,C12N 15/00 (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
Applicants:
CHEN PLOTKIN, Alice [US/US]; US
AMADO, Defne [US/US]; US
REIDERS, Julianne [US/US]; US
THE CHILDREN'S HOSPITAL OF PHILADELPHIA [US/US]; 3401 Civic Center Boulevard Philadelphia, Pennsylvania 19104, US
THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA [US/US]; 3160 Chestnut Street, Suite 200 Philadelphia, Pennsylvania 19104, US
Inventors:
CHEN PLOTKIN, Alice; US
AMADO, Defne; US
REIDERS, Julianne; US
DAVIDSON, Beverly L.; US
Agent:
BEDGOOD, Robert; US
Priority Data:
62/302,52502.03.2016US
Title (EN) THERAPY FOR FRONTOTEMPORAL DEMENTIA
(FR) THÉRAPIE POUR DÉMENCE FRONTOTEMPORALE
Abstract:
(EN) The invention provides methods and uses for delivering progranulin to the central nervous system (CNS) of a mammal. Methods and uses include, for example, administering to a mammal a vector comprising a nucleic acid encoding progranulin, variant, derivative or functional fragment thereof to the mammal's brain ventricle to transduce CNS cells and/or cells that contact the cerebrospinal fluid (CSF) of the mammal such that the cells express the progranulin, variant, derivative or functional fragment thereof.
(FR) La présente invention concerne des procédés et des utilisations pour administrer de la progranuline au niveau du système nerveux central (SNC) d'un mammifère. Les procédés et les utilisations comprennent, par exemple, l'administration à un mammifère d'un vecteur comprenant un acide nucléique codant la progranuline, une variante, un dérivé ou un fragment fonctionnel de celle-ci au ventricule cérébral du mammifère pour transduire des cellules du SNC et/ou des cellules qui entrent en contact avec le fluide cérébrospinal (CSF) du mammifère de sorte que les cellules expriment la progranuline, la variante, le dérivé ou le fragment fonctionnel de celle-ci.
front page image
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)