Targeted delivery through a cationic amino acid transporter

Details Targeted delivery through a cationic amino acid transporter Targeted delivery through a cationic amino acid transporter

C - Chemistry, Metallurgy

12

N

C12N 15/87 (2006.01) A61K 48/00 (2006.01) C07K 14/075 (2006.01) C07K 14/15 (2006.01) C07K 16/18 (2006.01) C12N 7/04 (2006.01) C12N 15/10 (2006.01) C12N 15/86 (2006.01) C12N 15/861 (2006.01) C12N 15/867 (2006.01)

Patent

CA 2329270

The invention relates to the targeted delivery of substances to cells. The invention provides a virus-like particle or gene delivery vehicle provided with a ligand capable of binding to a human amino acid transporter. Provided are for example ligands that can bind to the human transporter of cationic L- amino acids (hCAT1). Such hCAT1 binding molecules find applications in the design of vector systems for entry into human or primate cells. Preferred are retroviral envelope molecules, which - when incorporated in a virus particle - can infect hCAT1 positive cells at high frequencies. Also within the scope of the invention are methods for the design of such hCAT1 binding molecules.

L'invention se rapporte à l'administration ciblée de substances à des cellules. Elle concerne notamment une particule de type virus ou un véhicule d'administration génique doté d'un ligand susceptible de se lier à un transporteur d'acide aminé humain. Les ligands adaptés sont ceux qui se lient au transporteur humain des acides L-aminés cationiques (hCAT1). Ces molécules de liaison à hCAT1 s'avèrent utiles pour la conception de systèmes de vecteurs destinés à être introduits dans des cellules humaines ou de primates. Les molécules préférées sont des molécules enveloppes rétrovirales qui, lorsqu'elles sont incorporées à une particule virale, peuvent infecter des cellules positives hCAT1 à des fréquences élevées. L'invention se rapporte également à des méthodes de conception de telles molécules de liaison à hCAT1.

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