Some content of this application is unavailable at the moment.
If this situation persist, please contact us atFeedback&Contact
1. (WO1998049350) $i(IN VIVO) USE OF RECOMBINAGENIC OLIGONUCLEOBASES TO CORRECT GENETIC LESIONS IN HEPATOCYTES
Latest bibliographic data on file with the International Bureau

Pub. No.: WO/1998/049350 International Application No.: PCT/US1998/008834
Publication Date: 05.11.1998 International Filing Date: 30.04.1998
Chapter 2 Demand Filed: 24.11.1998
IPC:
A61K 9/107 (2006.01) ,A61K 9/127 (2006.01) ,C12N 15/10 (2006.01)
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
9
Medicinal preparations characterised by special physical form
10
Dispersions; Emulsions
107
Emulsions
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
9
Medicinal preparations characterised by special physical form
10
Dispersions; Emulsions
127
Liposomes
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
10
Processes for the isolation, preparation or purification of DNA or RNA
Applicants:
REGENTS OF THE UNIVERSITY OF MINNESOTA [US/US]; 1100 Washington Avenue South Minneapolis, MN 55415-1226, US (AL, AM, AT, AU, AZ, BA, BB, BE, BF, BG, BJ, BR, BY, CA, CF, CG, CH, CI, CM, CN, CU, CY, CZ, DE, DK, EE, ES, FI, FR, GA, GB, GE, GH, GM, GN, GR, HU, IE, IL, IS, IT, JP, KE, KG, KP, KR, KZ, LC, LK, LR, LS, LT, LU, LV, MC, MD, MG, MK, ML, MN, MR, MW, MX, NE, NL, NO, NZ, PL, PT, RO, RU, SD, SE, SG, SI, SK, SL, SN, SZ, TD, TG, TJ, TM, TR, TT, UA, UG, UZ, VN, YU, ZW)
STEER, Clifford, J. [US/US]; US (UsOnly)
KREN, Betsy, T. [US/US]; US (UsOnly)
BANDYOPADHYAY, Paramita, T. [IN/US]; US (UsOnly)
Inventors:
STEER, Clifford, J.; US
KREN, Betsy, T.; US
BANDYOPADHYAY, Paramita, T.; US
Agent:
HANSBURG, Daniel; Kimeragen, Inc. 300 Pheasant Run Newtown, PA 18940 , US
Priority Data:
60/045,28830.04.1997US
60/054,83705.08.1997US
60/064,99610.11.1997US
Title (EN) $i(IN VIVO) USE OF RECOMBINAGENIC OLIGONUCLEOBASES TO CORRECT GENETIC LESIONS IN HEPATOCYTES
(FR) UTILISATION $i(IN VIVO) D'OLIGONUCLEOBASES RECOMBINAGENES POUR CORRIGER LES LESIONS GENETIQUES DES HEPATOCYTES
Abstract:
(EN) The present invention concerns compositions and methods for the introduction of specific genetic changes in endogenous genes of the cells of an animal. The genetic changes are effected by oligonucleotides or oligonucleotide derivatives and analogs, which are generally less than about 100 nucleotides in length. The invention provides for macromolecular carriers, optionally incorporating ligands for clathrin coated pit receptors. In one embodiment the ligand is a lactose or galactose and the genetic changes are made in hepatocytes. By means of the invention up to 40 % of the copiies of a target gene have been changed $i(in vitro). Repair of mutant genes having a Crigler-Najjar like phenotype and Hemophilia B phenotype were observed.
(FR) L'invention porte sur des compositions et procédés d'introduction de modifications génétiques spécifiques dans des gènes endogènes des cellules d'un animal. Lesdites modifications sont opérées par des oligonucléotides ou leurs dérivés ou analogues normalement d'une longueur inférieure à environ 100 nucléotides. L'invention porte également sur des porteurs macromoléculaires comportant facultativement des ligands de 'pit receptors' à revêtement de clathrine. Dans l'une des exécutions, le ligand est du lactose ou du galactose et les modifications génétiques sont opérées par des hépatocytes. Cette invention a permis de modifier jusqu'à 40 % des copies d'un gène cible $i(in vitro). On a pu observer des réparations de gènes mutants présentant un phénotype du genre Crigler-Najjar et un phénotype du genre Hemophilia B.
Designated States: AL, AM, AU, BA, BB, BG, BR, CA, CN, CU, CZ, EE, GE, HU, IL, IS, JP, KP, KR, LC, LK, LR, LT, LV, MG, MK, MN, MX, NO, NZ, PL, RO, SG, SI, SK, SL, TR, TT, UA, US, UZ, VN, YU
African Regional Intellectual Property Organization (ARIPO) (GH, GM, KE, LS, MW, SD, SZ, UG, ZW)
Eurasian Patent Organization (AM, AZ, BY, KG, KZ, MD, RU, TJ, TM)
European Patent Office (AT, BE, CH, CY, DE, DK, ES, FI, FR, GB, GR, IE, IT, LU, MC, NL, PT, SE)
African Intellectual Property Organization (BF, BJ, CF, CG, CI, CM, GA, GN, ML, MR, NE, SN, TD, TG)
Publication Language: English (EN)
Filing Language: English (EN)
Also published as:
KR1020010020375EP0979311JP2002511851 CN1268186CA2288209NZ500694
AU1998073654