Polynucleotides Encoding Low Density Lipoprotein Receptor

摘要

The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotide molecules encoding low density lipoprotein receptor comprising at least one mutation (e.g., an LDLR signally enhancing mutation).

主权项

1. A polynucleotide encoding a low density lipoprotein receptor (LDLR) wherein the LDLR comprises at least one mutation in a domain selected from the group consisting of EGF-A domain, the intracellular domain and both the EGF-domain and the intracellular domain, wherein the polynucleotide comprises;
(a) a first region of linked nucleosides, said first region encoding a polypeptide of interest, said polypeptide of interest selected from the group consisting of SEQ ID NOs 37-55 and 724-729; (b) a first flanking region located 5′ relative to said first region comprising at least one 5′ terminal cap; (c) a second flanking region located 3′ relative to said first region comprising a 3′ tailing sequence of linked nucleosides; andwherein said polynucleotide comprises at least one chemically modified nucleoside.