Ionis Pharmaceuticals, Inc.

University of California

Ludwig Institute for Cancer Research Limited

Isis Pharmaceuticals, Inc.

Research Institute at Nationwide Children's Hospital

Ludwig Institute for Cancer

Don W Cleveland

Susan M Freier

C Frank Bennett

Seng H Cheng

Gene Hung

Lamya S Shihabuddin

Holly Kordasiewicz

Lisa Stanek

Roman Sakowicz

Kenneth Wood

Lawrence S B Goldstein

Frank Rigo

Clotilde Lagier-Tourenne

John M Ravits

Michael W Baughn

Kenneth W Dobie

Brian K Kaspar

Huynh-Hoa Bui

Ravi Jain

Richard Alan Smith

Thomas P Condon

Kevin Foust

Ze'ev Melamed

C Frank Bennet

Lamya Shihabuddin

Qiang Zhu

Jie Jiang

Clotilde Lagier-Tourene

Provided herein are methods, compounds, and compositions for reducing expression of huntingtin mRNA and protein in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate Huntington's disease, or a symptom thereof.

Modulation of huntingtin expression

Provided are compounds, methods, and pharmaceutical compositions for increasing the amount or activity of STMN2 RNA in a cell or animal, and in certain embodiments increasing the amount of STMN2 protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom of a neurodegenerative disease. Such symptoms include ataxia, neuropathy, synaptic dysfunction, deficits in cognition, and decreased longevity. Such neurodegenerative diseases include amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), Alzheimer's disease (AD), and dementia with Lewy bodies (DLB).

Compounds and methods for increasing STMN2 expression

Modulation of Huntington expression

Disclosed herein are compositions and methods for reducing expression of C9ORF72 antisense transcript in an animal with C9ORF72 antisense transcript specific inhibitors. Such methods are useful to treat, prevent, or ameliorate neurodegenerative diseases in an individual in need thereof. Such C9ORF72 antisense transcript specific inhibitors include antisense compounds.

Methods for modulating expression of C9ORF72 antisense transcript

The present invention relates to RNA-based methods for inhibiting the expression of the superoxide dismutase 1 (SOD-1) gene. Recombinant adeno-associated viruses of the invention deliver DNAs encoding RNAs that knock down the expression of SOD-1. The methods have application in the treatment of amyotrophic lateral sclerosis.

Products and methods for treatment of familial amyotrophic lateral sclerosis

Disclosed herein are methods for reducing expression of C90RF72 mRNA and protein in an animal with C90RF72 specific inhibitors. Such methods are useful to treat, prevent, or ameliorate neurodegenerative diseases in an individual in need thereof. Such C90RF72 specific inhibitors include antisense compounds. Examples of neurodegenerative diseases that can be treated, prevented, and ameliorated with the administration C90RF72 specific inhibitors include amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), corticalbasal degeneration syndrome (CBD), atypical Parkinsonian syndrome, and olivopontocerellar degeneration (OPCD).

Methods for modulating C9ORF72 expression

Antisense compounds, compositions and methods are provided for modulating the expression of superoxide dismutase 1, soluble. The compositions comprise antisense compounds, particularly antisense oligonucleotides, targeted to nucleic acids encoding superoxide dismutase 1, soluble. Methods of using these compounds for modulation of superoxide dismutase 1, soluble expression and for treatment of diseases associated with expression of superoxide dismutase 1, soluble are provided.

Antisense modulation of superoxide dismutase 1, soluble expression

The invention provides isolated nucleic acid and amino acid sequences ofCENP-E (XCENP-E), antibodies to XCENP-E, methods of screening for CENP-E modulators using biologically active CENP-E, and kits for screening for CENP-E modulators.

Plus end-directed microtubule motor required for chromosome congression

The invention provides isolated nucleic acid and amino acid sequences of Xenopus CENP-E (XCENP-E), antibodies to XCENP-E, methods of screening for CENP-E modulators using biologically active CENP-E, and kits for screening for CENP-E modulators.

Growing community of inventors

Del Mar, CA, United States of America

Don W Cleveland