Signal for targeting molecules to the sarco(endo)plasmic reticulum
AssigneeIntrexon Corporation
InventorThomas D. Reed
Life of this patent
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Abstract
The invention relates to targeting molecules to subcellular locations. In particular, the invention relates to compositions and methods to target molecules to the sarco(endo)plasmic reticulum (SR). The invention includes uses for polypeptide sequences as a localization signal to target therapeutic and experimental compounds or macromolecules to the SR of eukaryotic cells. The invention also includes nucleotide sequences encoding the polypeptide SR localization signal, allowing it to be incorporated into vectors for delivery of therapeutic or experimental gene products via in vitro or in vivo gene transfer.
Claims20
An isolated polypeptide comprising the amino acid sequence of SEQ ID NO:1 with at least two amino acid substitutions at two locations selected from the group consisting of Leu-9, Asn-12, Phe-13, Ile-16, Leu-20, Ile-26, Val-27, and Leu-30.
An isolated polypeptide comprising the amino acid sequence of SEQ ID NO:2.
An isolated nucleic acid comprising a nucleotide sequence encoding the polypeptide sequence of SEQ ID NO:1 with at least two codon substitutions encoding an amino acid substitution at two amino acid locations selected from the group consisting of Leu-9, Asn-12, Phe-13, Ile-16, Leu-20, Ile-26, Val-27, and Leu-30.
An isolated nucleic acid comprising a nucleotide sequence selected from nucleotide sequences represented by SEQ NO:3, SEQ ID NO:4, SEQ ID NO:5, and SEQ ID NO:6.
A method for targeting a compound or a macromolecule to the sarco(endo)plasmic region of a cell, the method comprising the steps of:
- (1) providing an isolated polypeptide comprising the amino acid sequence of SEQ ID NO:1 with at least one amino acid substitution at a location selected from the group consisting of Leu-9, Asn-12, Phe-13, Ile-16, Leu-20, Ile-26, Val-27, and Leu-30;
- (2) linking the polypeptide to a compound or macromolecule to provide a linked compound or linked macromolecule;
- (3) administering the polypeptide-linked compound or polypeptide-linked macromolecule to cells.
Dependencies are inferred from the claim text.