Isis Pharmaceuticals, Inc.

Ionis Pharmaceuticals, Inc.

Sanjay K Pandey

Susan M Freier

C Frank Bennett

Eric E Swayze

Sanjay Bhanot

Kenneth W Dobie

Robert McKay

Ravi Jain

Seng H Cheng

Eric G Marcusson

Robert A MacLeod

Murielle M Veniant-Ellison

Xing-Xian Yu

Richard A Lindberg

Bruce M Wentworth

Charles A Thornton

Thurman Wheeler

Andrew Leger

John R Shutter

Provided herein are methods, compounds, and compositions for reducing expression of a DMPK mRNA and protein in an animal. Also provided herein are methods, compounds, and compositions for preferentially reducing CUGexp DMPK RNA, reducing myotonia or reducing spliceopathy in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate type 1 myotonic dystrophy, or a symptom thereof.

Modulation of dystrophia myotonica-protein kinase (DMPK) expression

Antisense compounds, compositions and methods are provided for modulating the expression of forkhead box O1A. The compositions comprise antisense compounds, particularly antisense oligonucleotides, targeted to nucleic acids encoding forkhead box O1A. Methods of using these compounds for modulation of forkhead box O1A expression and for treatment of diseases associated with expression of forkhead box O1A are provided, in particular, for methods of treating diabetes.

Modulation of forkhead box O1A expression

Disclosed herein are compounds and methods for decreasing SMRT and treating metabolic and/or cardiovascular diseases in an individual in need thereof. Examples of disease conditions that can be ameliorated with the administration of antisense compounds targeted to SMRT include obesity, diabetes, dyslipidemia, and hypothyroidism.

Modulation of SMRT expression

Disclosed herein are compounds, compositions and methods for modulating the expression of PTPRU in a cell, tissue or animal. Also provided are methods of active target segment validation. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders. Also provided are methods for the prevention, amelioration and/or treatment of diabetes, obesity, insulin resistance, insulin deficiency, hypercholesterolemia, hyperglycemia, hyperlipidemia, hypertriglyceridemia, hyperfattyacidemia, liver steatosis, steatohepatitis, non-alcoholic steatohepatitis, metabolic syndrome, cardiovascular disease and coronary heart disease by administration of antisense compounds targeted to PTPRU.

Compositions and their uses directed to PTPRU

Growing community of inventors

Encinitas, CA, United States of America

Sanjay K Pandey