A - Human Necessities – 61 – K
Patent
A - Human Necessities
61
K
A61K 48/00 (2006.01) A61K 9/127 (2006.01) A61K 31/70 (2006.01) A61K 38/17 (2006.01) A61K 47/22 (2006.01) A61K 47/24 (2006.01) A61K 47/28 (2006.01) A61K 47/48 (2006.01) C12N 15/88 (2006.01)
Patent
CA 2223923
The invention herein describes pharmaceutical compositions and methods for targeted delivery of functional genes into cells and tissues in vivo. The invention discloses DNA:lipid complexes, methods of making such complexes and methods of using such complexes for facilitating the targeted delivery and entry of recombinant expression constructs into cells and tissues in vivo, and particularly delivery of such recombinant expression constructs into cells and tissues in vivo, and particularly delivery of such recombinant expression constructs by intravenous, intraperitoneal or direct injection. The delivery vehicle for the targeting of the recombinant construct encoding the gene of interest, is composed of a mixture of a cationic imidazolinium lipid of formula (I) wherein R and R1 mean a C11-29 aliphatic hydrocarbyl group, together with neutral lipids (e.g. DOPE, cholesterol).
La présente invention concerne des compositions et des procédés pharmaceutiques permettant un apport ciblé in vivo de gènes fonctionnels dans des cellules et des tissus. En l'occurrence, l'invention concerne des complexes ADN/lipide, des procédés de production de tels complexes, ainsi que des procédés d'utilisation de ces complexes pour faciliter l'apport et l'introduction ciblés de produits d'assemblage d'expression recombinés in vivo dans des cellules et tissus, et notamment l'apport de tels produits d'assemblage d'expression recombinés par injection intraveineuse, intrapéritonéale ou directe. Le véhicule d'apport du produit d'assemblage recombiné codant le gène d'intérêt se compose d'un mélange d'un lipide d'imidazolinium cationique de formule (I), dans laquelle R et R¿1? représentent un groupe hydrocarbyle aliphatique C¿11-29?, avec des lipides neutres (par exemple, DOPE, cholestérol).
Gorman Cori M.
Mcclarrinon Molly
Mbm & Co.
Megabios Corporation
Valentis Inc.
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