Principal Investigators

    MILLER, TIMOTHY M

    Institution

    WASHINGTON UNIVERSITY

    Contact information of lead PI

    Country

    USA

    Title of project or programme

    DEVELOPING A MICRORNA-TARGETED THERAPY FOR ALS

    Source of funding information

    NIH (NINDS)

    Total sum awarded (Euro)

    305045.8716

    Start date of award

    15/04/2012

    Total duration of award in years

    1

    Keywords

    Amyotrophic Lateral Sclerosis, Antisense Oligonucleotides, targeted treatment, MicroRNAs, Spinal Cord

    Research Abstract

    DESCRIPTION (provided by applicant): Amyotrophic lateral sclerosis is characterized by the progressive loss of motor neurons in the spinal cord, resulting in stiffness, severe weakness, atrophy of skeletal muscles, and eventual death from respiratory failure in 3-5 years. There are no current therapies that substantially slow the progression of the disease. In animal models and in samples from ALS patients, we have discovered changes in small non-coding RNA called microRNAs. We will now validate one particular microRNA as a therapeutic target and develop a method of inhibiting this microRNA using antisense oligonucleotides. We hypothesize that inhibition of this miRNA will substantially slow ALS in animal models. Given our current experience in Phase I trial using antisense oligonucleotides in ALS patients; we intend to translate our findings from this grant to a novel therapeutic for ALS.

    Further information available at:

Types: Investments < €500k
Member States: United States of America
Diseases: N/A
Years: 2016
Database Categories: N/A
Database Tags: N/A

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