A - Human Necessities – 61 – K
Patent
A - Human Necessities
61
K
A61K 9/127 (2006.01) A61K 39/44 (2006.01) A61K 47/48 (2006.01) A61K 48/00 (2006.01) C12N 15/63 (2006.01) C12N 15/88 (2006.01)
Patent
CA 2335638
The effective introduction of foreign genes and other biologically active molecules into targeted mammalian cells is a challenge still facing those skilled in the art. Gene therapy, for example, requires successful transfection of target cells in a patient. The present invention relates to novel micellar complexes of cationic amphiphilic compounds that facilitate delivery of biologically active molecules to the targeted cells of a mammal. The novel micellar complexes are comprised of a cationic amphiphile, a biologically active molecule, a derivative of polyethylene glycol (PEG), and optionally, a co-lipid. A further aspect of the invention is the use of targeting absents in any of the methods that effectuate the delivery of biologically active molecules into the cells of mammals. A targeting agent is usually any molecule, peptide sequence, or large protein that preferentially targets or binds to specific mammalian cells.
L'introduction efficace de gènes étrangers et autres molécules biologiquement actives dans des cellules cible de mammifères reste un défi pour les scientifiques de la profession. La thérapie génique, par exemple, nécessite la transfection réussie de cellules cible chez un patient. La présente invention concerne de nouveaux complexes micellaires de composés amphiphiles cationiques favorisant l'apport de molécules biologiquement actives aux cellules cibles chez un mammifère. Ces nouveaux complexes micellaires comprennent un amphiphile cationique, une molécule biologiquement active, un dérivé du polyéthylène glycol, et éventuellement, un co-lipide. Un autre aspect de l'invention concerne l'utilisation d'agents cible dans n'importe quel procédé permettant l'apport de molécules biologiquement actives dans les cellules de mammifères. Un agent cible est, de façon générale, n'importe quelle molécule, séquence peptidique ou protéine de grande taille ciblant ou se liant de préférence à des cellules de mammifères spécifiques.
Cheng Seng H.
Chu Quiming
Eastman Simon J.
Lee Edward R.
Marshall John
Blake Cassels & Graydon Llp
Genzyme Corporation
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