Website Search
ID 16947

Small Molecule Therapy for SMA

Dr. Charlotte Sumner describes small molecule therapies for SMA.
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.
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
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.

Related content:

16950. 2D Animation of Antisense Oligonucleotide Therapy for SMA
An animation shows how antisense oligonucleotide therapy for SMA utilizes RNA splicing.
16948. Alternative RNA Splicing Therapy for Spinal Muscular Atrophy
Drs. Sharp and Sumner describe how RNA splicing can be used as a therapy for SMA.
16941. 2D Animation of Alternative RNA Splicing
An animation shows alternate splicing of the SMN2 gene.
16910. Cause of SMA
Dr. Sumner explains what causes SMA.
16946. An Overview of Future Therapies for SMA
Dr. Sumner provides an overview of targets for SMA therapies.
16949. Antisense Oligonucletotide Therapy for SMA
Dr. Krainer explains the science behind antisense therapy for SMA.
16935. The Connection Between SMA and RNA Splicing
Dr. Krainer explains the connection between SMA and RNA splicing.
16938. 3D Animation of RNA Splicing
An animation of the crucial RNA editing step called splicing.
16953. Gene Therapy for SMA
Dr. Sumner talks about gene therapy for SMA.
16929. Genes Involved with SMA
Dr. Sumner explains how SMA is related to changes in the SMN1 and SMN2 genes.
Cold Spring Harbor Laboratory
CSHL HomeAbout CSHLResearchEducationPublic EventsNewsstandPartner With UsGiving