C - Chemistry – Metallurgy – 12 – Q
Patent
C - Chemistry, Metallurgy
12
Q
C12Q 1/68 (2006.01) A61K 31/70 (2006.01) A61K 47/48 (2006.01) A61K 48/00 (2006.01) C07H 21/00 (2006.01) C07K 1/22 (2006.01) C07K 14/485 (2006.01) C07K 14/79 (2006.01) C07K 16/00 (2006.01) C12N 15/87 (2006.01)
Patent
CA 2079109
ABSTRACT OF THE DISCLOSURE The invention relates to compositions and methods for enhancing the delivery of an oligonucleotide into a cell. The compositions of the invention comprise oligonucleotide conjugates which consist of an oligonucleotide, conjugated via a molecular linker containing at least one disulfide bond, to an agent (termed herein "transport agent") which facilitates transport across an outer cell membrane, or across the blood-brain barrier. In a preferred aspect, the disulfide linkage is cleaved upon uptake of the composition by the cell. Pharmaceutical compositions comprising an oligonucleotide conjugate of the invention may be used to treat a wide variety of diseases and disorders. Methods for inhibiting the expression of a nucleic acid sequence within a cell, and methods for detecting a nucleic acid sequence within a cell are also provided. In a specific embodiment, an oligonucleotide conjugated to cholesterol via a linker containing a disulfide linkage can be used for therapeutic or diagnostic purposes, by hybridization of the oligonucleotide to a complementary nucleic acid sequence in a procaryotic or eucaryotic cell.
Latham John A.
Lin Kuei-Ying
Matteucci Mark
Gilead Sciences Inc.
Osler Hoskin & Harcourt Llp
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