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ID 16953

Gene Therapy for SMA

Description:
Dr. Charlotte Sumner talks about gene therapy for SMA.
Transcript:
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.
Keywords:
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
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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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