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ID 16953

Gene Therapy for SMA

Dr. Charlotte Sumner talks about gene therapy for SMA.
Another broad category of treatment of course is to try and put back what's missing. So, instead of trying to alter SMN2 could we actually just put back an SMN1 gene. And of course that is now getting into the realm gene therapy, which we've been working for many decades in numerous kinds of inherited disorders, genetic disorders and there have been challenges to gene therapy. The principal one has been trying to get a desired gene where you want it and get it there safely in such a way that it doesn't create new problems. And that's been difficult and could be particularly challenging for motor neuron disease where the motor neuron that you're trying to target, it's cell body is in the spinal cord. That's a very hard place to access and get a gene into that particular cell. But recently, there's been enormous success by several laboratories around the country and really the success stems of identifying new vectors, new packages for delivering these genes.
Spinal muscular atrophy, SMA, RNA, mRNA, splicing, gene, genetic, DNA, antisense, motor neuron, splice, Therapy, SMN1, SMN2, stem cell therapy, germ cell, vector, inherit, gene replacement, Hopkins, john Hopkins, 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.

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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.
16946. An Overview of Future Therapies for SMA
Dr. Sumner provides an overview of targets for SMA therapies.
16950. 2D Animation of Antisense Oligonucleotide Therapy for SMA
An animation shows how antisense oligonucleotide therapy for SMA utilizes RNA splicing.
16910. Cause of SMA
Dr. Sumner explains what causes SMA.
16929. Genes Involved with SMA
Dr. Sumner explains how SMA is related to changes in the SMN1 and SMN2 genes.
16911. A simple description of the cause of SMA
Frank, an SMA parent, describes the genetics of SMA.
16941. 2D Animation of Alternative RNA Splicing
An animation shows alternate splicing of the SMN2 gene.
16934. Gene Copy Number Variation in SMA
Dr. Sumner explains copy number variation of the SMN2 gene may influence disease severity.
16949. Antisense Oligonucletotide Therapy for SMA
Dr. Krainer explains the science behind antisense therapy for SMA.
16947. Small Molecule Therapy for SMA
Dr. Sumner describes small molecule therapies for SMA.
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