Sparky RNA repair

RNA repair: "The researchers, led by Al George, used a dog model of myotonia congenita, a non-lethal condition that is characterized by muscle stiffness and caused by mutations in the chloride channels of skeletal muscle cells. Targeting a mutation discovered in an affected dog, they engineered a ribozyme that splices off the mutation-containing 3? section of the mRNA and replaces it with the wild-type sequence. This technique is called ribozyme-mediated trans-splicing. The ribozyme is produced as a DNA template, which is transfected into cells using a vector, in the same way as in standard gene therapy. The template is then transcribed within the cell to create the RNA ribozyme. In theory, the ribozyme is continuously transcribed and is over expressed, which should mean that enough is present to repair all mutant mRNA in the cell.

George's group used a plasmid vector to transfect their ribozyme into a cell line with impaired chloride transportation caused by the mutation, which was named Sparky after the dog in which it was identified.

These results offer the first direct proof that ribozymes can repair mutant mRNA to produce functional wild-type proteins."