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Families of SMA Awards $150,000 to Investigate New Antisense Therapies for Spinal Muscular Atrophy
Date:5/7/2013

Elk Grove Village, IL (PRWEB) May 07, 2013

Families of SMA is dedicated to creating a treatment and cure for Spinal Muscular Atrophy (SMA) by funding and advancing a comprehensive research program, including drug discovery programs to make practical new therapies. This is the third drug discovery project awarded by Families of SMA in 2013 with a total investment of $400,000. This program will assess the therapeutic potential of second-generation Antisense Oligonucleotides (ASO) sequences for SMA. This funding is being awarded to Co-Principal Investigators Dr. Arthur Burghes at Ohio State University and Dr. Christian Lorson at University of Missouri.

Program Description: New Morpholino Antisense Oligonucleotides for the Treatment of SMA.

Objective: Antisense oligonucleotides (ASOs) targeted to the ISS-N1 region have been shown to be effective in mice and are now being tested in the current clinical trials conducted by Isis Pharmaceuticals. The major goal of this new program is to explore the therapeutic potential of two new sequences, which are located in different regions of the SMN2 gene. Importantly, the possibility exists to use these different ASOs in combination. The ASOs in this project will be generated using a new morpholino backbone chemistry.

Research Strategy: First, the Burghes and Lorson groups will determine the optimal nucleic acid sequence. They will then establish the best dose of the optimized ASOs to provide maximal rescue in the severe Delta7 mouse model of SMA, developed by Dr. Burghes. Knowing this information in mice will help design subsequent studies in primates to assess whether these ASOs reach the correct tissues including motor neurons, when delivered into the cerebral spinal fluid (CSF) in larger animals. Dr. Ravindra Singh at Iowa State University will be collaborating on the project.

How Does the ASO Approach for SMA Work?
This therapeutic approach fo
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