The Connection Between SMA and RNA Splicing

Researcher Dr. Adrian Krainer explains the connection between SMA and RNA splicing.

So the connection between splicing and SMA is actually twofold. You know, one has to do with the function of the SMN protein itself or one of its functions, which is to assemble components that are critical for splicing in general. And the second connection has to do with splicing of the SMN2 gene itself. Because it is alternatively spliced in a way that gives a reduced amount of functional SMN protein, if it's possible to target that gene and change its splicing to give more full length SMN protein that would make it a more functional gene.

Spinal muscular atrophy, SMA, RNA, mRNA, splicing, gene, genetic, DNA, antisense, motor neuron, splice, Transcription, intron, exon, pre mRNA, splicing, spliceosome, function, alternative splicing, binding site, exon 7

Related Content

16941. 2D Animation of Alternative RNA Splicing

An animation shows alternate splicing of the SMN2 gene.

  • ID: 16941
  • Source: DNALC.SMA

16939. RNA Splicing

A step-by-step 2D animation shows the details of RNA splicing.

  • ID: 16939
  • Source: DNALC.SMA

16937. An Explanation of RNA Splicing

Dr. Sharp explains the process of RNA splicing.

  • ID: 16937
  • Source: DNALC.SMA

16938. 3D Animation of RNA Splicing

An animation of the crucial RNA editing step called splicing.

  • ID: 16938
  • Source: DNALC.SMA

16950. 2D Animation of Antisense Oligonucleotide Therapy for SMA

An animation shows how antisense oligonucleotide therapy for SMA utilizes RNA splicing.

  • ID: 16950
  • Source: DNALC.SMA

16940. Alternative RNA Splicing

Drs. Sharp and Krainer explain how genes can be alternatively spliced.

  • ID: 16940
  • Source: DNALC.SMA

16949. Antisense Oligonucletotide Therapy for SMA

Dr. Krainer explains the science behind antisense therapy for SMA.

  • ID: 16949
  • Source: DNALC.SMA

16936. An Introduction to RNA Splicing

Dr. Roberts describes RNA splicing.

  • ID: 16936
  • Source: DNALC.SMA

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.

  • ID: 16948
  • Source: DNALC.SMA

16929. Genes Involved with SMA

Dr. Sumner explains how SMA is related to changes in the SMN1 and SMN2 genes.

  • ID: 16929
  • Source: DNALC.SMA