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ID 16947

Small Molecule Therapy for SMA

Description:
Dr. Charlotte Sumner describes small molecule therapies for SMA.
Transcript:
Now, one of the basic types of strategies has been to try and find small molecules, drugs, that activate the expression of the gene at the gene level. So each gene is controlled in its expression pattern by upstream genetic sequence what we call a promoter and that promoter is sometimes more active, sometimes less active. But the idea of drugs to increase SMN expression at the gene level has been to try and find drugs that would activate the promoter of the gene. And there have been numerous efforts to find such molecules. Years ago, there was an observation that a kind of drug called an HDAC inhibitor, a histone deacetylase inhibitor could activate gene expression. The trouble with HDAC inhibitors in general is they're a fairly toxic kind of drug. Another drug that works in this same way presumably is what we call the quinazolines. So this is another drug that was discovered that could increase SMN expression at presumably the promoter level.
Keywords:
Spinal muscular atrophy, SMA, RNA, mRNA, splicing, gene, genetic, DNA, antisense, motor neuron, splice, Therapy, Molecule, expression, promoter, SMN, HDAC inhibitor, quinazolines, Hopkins, john Hopkins, transcription, exon 7
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Creative Commons License This work by Cold Spring Harbor Laboratory is licensed under a Creative Commons Attribution-Noncommercial-No Derivative Works 3.0 United States License.

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