Johns Hopkins University

The Salk Institute for Biological Studies

Ionis Pharmaceuticals, Inc.

Jeffrey D Rothstein

Mandy Jackson

Glen Lin

Robert Law

Irina Orlov

Susan M Freier

C Frank Bennett

Fred Harrison Gage

Brian K Kaspar

Daniel A Peterson

Christopher Donnelly

Chieng-Liang Glenn Lin

Rita G Sattler

Lynn A Bristol

Alicia Ruggiero

Disclosed herein are methods for monitoring expression of C9ORF72 mRNA and protein in an animal with C9ORF72 specific inhibitors. Such C9ORF72 specific inhibitors include antisense compounds.

Methods for monitoring C9ORF72 expression

The present invention provides methods and compositions for identifying compounds which modulate cellular glycosylation. The invention further provides methods for treating subjects suffering from or at risk of developing a glycosylation associated disorder.

Methods of identifying modulators of cellular glycosylation using GTRAP3-18

The present invention provides nucleic acid molecules comprising the EAAT2 promoter, as well as screening assays useful for identifying compounds which modulate the activity of the EAAT2 promoter, and methods of treating neurological disorders comprising administration of EAAT2 promoter modulators.

EAAT2 promoter and uses thereof

Methods are disclosed for delivering a heterologous gene to a cell body of a neuron by contacting a muscle tissue innervated by the neuron with a viral vector comprising a heterologous gene, wherein the viral vector enters said neuron and is retrogradely moved to the cell body. Additionally, methods for expressing secreted proteins from a nerve cell body as well as methods for treating neurodegenerative disorders such as amyotrophic lateral sclerosis are described.

Targeted retrograde gene delivery to motor neurons

Glutamate Transporter Associated Proteins and nucleotide encoding Glutamate Transporter Associated Proteins are provided. Also provided is a method for identifying a compound that modulates a cellular response mediated by a Glutamate Transporter Associated Protein. A method is further provided for identifying a compound that inhibits an interaction between a Glutamate Transporter Associated Protein and a glutamate transporter protein. A method is provided for treating a disorder associated with glutamate transport.

Glutamate transporter associated protein

Glutamate transporter associated proteins and methods of use thereof

In one aspect, the present invention features methods for detecting at least one neurological disorder in a patient, the method comprising obtaining a biological sample from the patient; and detecting at least one aberrant human glutamate transporter 2 (EAAT 2) mRNA in the sample as being indicative of the neurological disorder in the patient. In a particular aspect, the invention is useful for detecting amyotrophic lateral sclerosis (ALS) in the patient.

Aberrant glutamate transporters and methods of use

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Catonsville, MD, United States of America

Jeffrey D Rothstein