From reading the source doc https://mdaconference.org/node/1150 , the scientists put 4 modified snRNA molecules into a virus to deliver into the cell.
From reading the main article, 11% of DMS sufferers have an issue with duplicate exon 2s. From basic biochemistry, when the mRNA with the extra exon 2 gets translated by the ribosome into dystrophin, the resulting dystrophin protein doesn’t work.
The 4 snRNA molecules per the main article prevent the extra exon 2s from being connected into the mRNA resulting in an mRNA that gets translated into a functioning dystrophin protein.
So there is no modification of the DNA, only the resulting mRNA that was transcribed from the DNA (or gene) gets modified,
From reading the source doc https://mdaconference.org/node/1150 , the scientists put 4 modified snRNA molecules into a virus to deliver into the cell.
From reading the main article, 11% of DMS sufferers have an issue with duplicate exon 2s. From basic biochemistry, when the mRNA with the extra exon 2 gets translated by the ribosome into dystrophin, the resulting dystrophin protein doesn’t work.
The 4 snRNA molecules per the main article prevent the extra exon 2s from being connected into the mRNA resulting in an mRNA that gets translated into a functioning dystrophin protein.
So there is no modification of the DNA, only the resulting mRNA that was transcribed from the DNA (or gene) gets modified,