We are currently investigating platforms which combine a gene therapy-based approach with mitochondrial target sequence (MTS) and optogenetics. Read below for more information about these technologies.
Gene therapy
Genes are a set of genetic instructions for cells, many of which are used to make specific proteins.
Gene therapy involves the transfer of genes into cells. This can be used to either replace or remove faulty genes that cause a disease (e.g. because they fail to produce a protein that works), or to produce different proteins that may help.
Gene therapy offers the possibility of a treatment for certain diseases which can provide a long-term benefit.
Mitochondrial targeting sequence
GenSight has access to an MTS technology platform which delivers a mitochondrial gene using a type of genetic material called messenger ribonucleic acid, or mRNA, to the surface of mitochondria. Early research suggests that this may allow missing mitochondrial proteins affected in retinal diseases to be made and actively shuttled into the mitochondria, restoring their function.
Optogenetics
Optogenetics is a technique that involves the transfer of a gene which instructs cells to make light-sensitive proteins which can allow them to respond to light stimulation. Our platform of optogenetics targets cells in the retina (retinal ganglion cells (RGCs)) and changes them into light-sensitive photoreceptor cells.
We are currently investigating the potential of combining this technology with a medical device to deliver light at the right intensity and wavelength.
Diseases we target
learn more