C - Chemistry – Metallurgy – 12 – Q
Patent
C - Chemistry, Metallurgy
12
Q
C12Q 1/68 (2006.01) A61K 9/107 (2006.01) A61K 9/127 (2006.01) A61K 48/00 (2006.01) C07H 21/04 (2006.01) C12N 15/10 (2006.01)
Patent
CA 2288209
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 in vitro. Repair of mutant genes having a Crigler- Najjar like phenotype and Hemophilia B phenotype were observed.
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 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.
Bandy-Opadhyay Paramita T.
Kren Betsy T.
Steer Clifford J.
Osler Hoskin & Harcourt Llp
Regents Of The University Of Minnesota
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