コレステロールレベルを低下させるためのＬＤＬＲ変異体および組成物中でのそれらの使用 - The Trustees of the University of Pennsylvania

Title:

コレステロールレベルを低下させるためのＬＤＬＲ変異体および組成物中でのそれらの使用

Document Type and Number:

Japanese Patent JP6741590

Kind Code:

B2

Abstract:

A recombinant vector having an expression cassette comprising a modified human low density lipoprotein receptor (hLDLR) gene is provided, wherein said hLDLR gene encodes a modified hLDLR comprising (a) one or more of the following amino acid substitutions: L318H, N295D, H306D, V307D, N309A, D310N, L318H, and/or L318D; or (b) an amino acid substitution of any of (a) in combination with one or more of the following amino acid substitutions: K796, K809R and/or C818A. Also provided are pharmaceutical compositions containing this vector and uses therefor in lowering cholesterol and/or treating familial hypercholesterolemia.

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Inventors:

Wilson, James M
Ladder, Daniel Jei
Somanasan, Srian Rayan

Application Number:

JP2016564200A

Publication Date:

August 19, 2020

Filing Date:

April 24, 2015

Export Citation:

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Assignee:

The Trustees of the University of Pennsylvania

International Classes:

C12N15/63; A61K48/00; A61P3/06; C12N15/12

Other References:

Da-Wei Zhang et al.,Journal of Biological Chemistry,2007年,Vol.282, No.25,p.18602-18612
Hong-mei Gu et al.,Journal of Lipid Research,2013年,Vol.54,p.3345-3357
Faisal A Al-Allaf et al.,International Archives of Medicine,2010年,Vol.3, No.36,p.1-25
Noam Zelcer et al.,Science,2009年 7月 3日,Vol.325,p.100-104
William R Lagor et al.,Journal of Receptor, Ligand and Channel Research,2010年,Vol.3,p.1-14

Attorney, Agent or Firm:

Patent business corporation Odashima patent office

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