Drug Delivery Mechanisms for SMA
Dr. Charlotte Sumner explains how drugs to treat SMA need to be delivered directly to the central nervous system, bypassing the blood-brain barrier.
The big challenge often is that the nervous system has a barrier that prevents many substances from passing into it, the so-called blood brain barrier. And the blood brain barrier is enormously helpful to us actually because it prevents infectious organisms and things from accessing the nervous system and that's a good thing in general. But it makes it a real challenge at times when developing drugs or novel therapeutics because many of these don�€™t naturally pass across the blood brain barrier. Of course there are ways we can get around that. We can actually deliver therapeutics directly to the nervous system, but it is more challenging. One has to put pumps into the nervous system or other sorts of devices to bypass the blood brain barrier and that of course is a bigger challenge.
Spinal muscular atrophy, SMA, RNA, mRNA, splicing, gene, genetic, DNA, antisense, motor neuron, splice, Therapy, Intrathecal, delivery, drug, mechanism, central nervous system, spinal cord, epidural, inject, needle, cerebrospinal fluid, blood-brain barrier, vertebra, axon, dendrite, Hopkins, john Hopkins,
- ID: 16951
- Source: DNALC.SMA
- Download: MPEG 4 Video
An animation shows how drugs for SMA may be delivered via infusion into the spinal cord.
Dr. Sumner discusses the clinical trial process for SMA antisense oligonucleotide therapy.
An animation shows how antisense oligonucleotide therapy for SMA utilizes RNA splicing.
Dr. Sumner describes small molecule therapies for SMA.
Drs. Sharp and Sumner describe how RNA splicing can be used as a therapy for SMA.
An animation shows alternate splicing of the SMN2 gene.
Dr. Sumner describes how early intervention is crucial when treating SMA.
Dr. Sumner explains what causes SMA.
Dr. Sumner provides an overview of targets for SMA therapies.
Dr. Sumner talks about gene therapy for SMA.