What is the molecular patch or exon skipping technique?
It involves making a very small piece of genetic material (molecular patch), which once inside a muscle cell, will bind to its matching sequence of genetic code. This patch is designed so that it binds a region surrounding the genetic error. When editing takes place to remove the non-essential regions of code, the area covered by the patch is not included in the final sequence, which goes on to produce the protein. In this way the reading frame is altered so that it becomes readable. This may be clearer using the following example:Consider the mutation we used above where the letters ND are missing. If a patch is made to bind to the letters AHISDOG, and delivered to muscle cells, it will bind to its matching genetic sequence. During editing the AHISDOG will not be included and we are left with:The effect of this is to turn a sentence, which could not be read, into one, which can. With the dystrophin gene, this is the difference between no protein being produced and a BMD like protein b
