A new approach to treating inherited sight loss
Here at Retina UK, we are always on the lookout for exciting new approaches for treating inherited sight loss and this story caught out attention.
Epic Bio have developed a new approach using Epigenetic editing for rare diseases. They have called this platform the Gene Expression Modulation System (GEMS) which can reliably modify the epigenome. Epigenetic editing is a unique approach which can change the expression of genes without making any permanent changes to the gene itself.
The epigenome sits on top of the genetic code and controls the expression of genes in cells and determines what genes are expressed and at what levels.
Gene expression is the extent to which a gene is switched on or off. For example, how much the retinal cell reads and uses a certain gene to build the protein that the gene codes for. There are also some genes that are not expressed at al in certain cells.
Epigenetics as a therapeutic approach can change the activity of cells without affecting the DNA. GEMS work by first locating the exact gene then attaching different GEMS to precisely adjust the epigenome, fine-tuning how DNA is read.
This approach has the potential to treat diseases that are unable to be treated using traditional gene therapies. Epic Bio is developing this technology for various conditions, one of them being Retinitis pigmentosa type 4 (RP4) which is caused by mutations in the RHO gene.
We will keep you updated as more developments come.