The University of British Columbia

Xenon Pharmaceuticals Inc.

Wisconsin Alumni Research Foundation

Other

Xenon Genetics Inc.

Isis Pharmaceuticals, Inc.

Hsc Research and Development Limited

Aspreva Pharmaceuticals S.a.

Aspreva Pharmaceuticals Corp.

Ionis Pharmaceuticals, Inc.

Michael R Hayden

Simon N Pimstone

Alan D Attie

Mark Gray-Keller

James M Ntambi

Alison J Brownlie

Makoto Miyazaki

Angela R Brooks-Wilson

Jeffrey Carroll

Simon Warby

Yuanhong Ma

Angela R Brooks-Wiison

Amber L Southwell

Christopher Kay

Suzanne Lewis

Guoquing Liu

Nicholas S Caron

Paul Goldberg

Susan M Freier

C Frank Bennett

Eric E Swayze

Mark Eric Cook

Johanna Rommens

Michel Perricaudet

Patrice Denefle

Patrick Benoit

Vikramjit S Chopra

Sarah Greenlee

Susan Andrew

Jamal Nasir

Biaoyang Lin

John P Kastelein

M E Suzanne Lewis

Richard Glickman

Michael Kalchman

Noel Hall

Abigail Hackam

Mark P Gray-Keller

The present invention relates to compositions and methods of use thereof for inhibiting mutant HTT mRNA transcription or CAG-expanded HTT protein expression in a cell, comprising contacting the cell with an effective amount of an oligomer targeting a differentiating polymorphism, wherein the differentiating polymorphism is selected from rs72239206, rs363107, rs362313, rs2530595, rs113407847. Specific oligomer sequences are also provided.

Allele-specific therapy for Huntington disease haplotypes

Allele-specific therapy for huntington disease haplotypes

The invention provides for the use of a therapeutic derived from a truncated lipoprotein lipase protein (LPL S447X), including nucleic acids encoding such proteins, for the treatment of conditions including LPL responsive conditions, such as cardiovascular disease, hypertension, LPL deficiency, high triglyceride levels, low HDL-cholesterol levels or atherosclerosis.

LPL variant therapeutics

Methods and compositions for reducing expression of a mutant huntingtin (mHTT) protein in a cell are provided. Such methods include contacting the cell with an effective amount of a nucleic acid silencing agent targeting a differentiating polymorphism in RNA encoding the mHTT.

Methods and compositions for the treatment of huntington's disease

Disclosed herein are antisense compounds and methods for selectively reducing expression of an allelic variant of a huntingtin gene containing a single nucleotide polymorphism (SNP). Such methods, compounds, and composition are useful to treat, prevent, or ameliorate Huntington's Disease (HD).

Selective reduction of allelic variants

The disclosure provides methods and compositions for treating a vascular, autoimmune, and/or inflammatory disease, or a condition associated therewith, with a dose of an inosine monophosphate dehydrogenase (IMPDH) inhibitor effective to treat the vascular, autoimmune and/or inflammatory disease, or associated condition but that does not produce immunosuppression sufficient to reduce the risk of allograft rejection. These lower doses of the immunosuppressant avoid the adverse consequences of severe immunosuppression while providing effective treatment of the vascular, autoimmune and/or inflammatory disease.

Treatment of vascular, autoimmune and inflammatory diseases using low dosages of IMPDH inhibitors

The invention features ABC1 nucleic acids and polypeptides for the diagnosis and treatment of abnormal cholesterol regulation. The invention also features methods for identifying compounds for modulating cholesterol levels in an animal (e.g., a human).

Methods and reagents for modulating cholesterol levels

Methods and compositions for the treatment of Huntington'S disease

Polynucleotide encoding an ATP binding cassette transporter 1 (ABC1) polypeptide

The use of screening assays based on the role of human stearoyl-CoA desaturase-1 ('hSCD1') in human diseases, disorders or conditions relating to serum levels of triglyceride, VLDL, HDL, LDL, total cholesterol, or production of secretions from mucous membranes, monounsaturated fatty acids, wax esters, and the like, is disclosed. Also disclosed are conventions useful in the prevention and/or treatment of such diseases.

Methods and compositions using stearoyl-CoA desaturase to identify triglyceride reducing therapeutic agents

Methods and compounds are disclosed for lowering serum LDL levels or serum cholesterol levels, or for reducing the transport of cholesterol from the gut to the blood or the lymph, based on the observation that a gene known as ABC1 is necessary in order for cholesterol to be transported from the intestinal lumen into the bloodstream. A mutant chicken phenotype, known as the WHAM chicken, characterized by low levels of serum LDL and reduced transport of cholesterol, facilitated the discovery of this function of the ABC1 gene. Techniques which act to inhibit ABC1 activity in the cells of the intestinal wall will result in lower serum cholesterol.

Cholesterol transport gene

Recombinant viruses comprising a heterologous DNA sequence coding for a lipase involved in lipoprotein metabolism. The invention also concerns the preparation and use in therapy of said recombinant viruses, especially for the treatment or prevention of dyslipoproteinemia-related pathologies.

Recombinant viruses and their use for treatment of atherosclerosis and other forms of coronary artery disease and method, reagent, and kit for evaluating susceptibility to same

Growing community of inventors

Vancouver, Canada

Michael R Hayden