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.

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