Genes and Gene Therapy
The theory behind gene therapy is to treat the disease by repairing the abnormal gene. This is achieved by replacing the disease-causing faulty gene with a “normal” copy into an individual’s cells. The most successful method to deliver the gene to the cells is by using a harmless virus that has been genetically modified to carry human DNA. The eye has proved to be an ideal organ for gene therapy as it is well protected from the body’s immune response. These early successes are paving the way in the near future for treatments for both inherited and non-inherited forms of blindness. Click here to read more about genes and gene therapy.
Cell Therapy and Regenerative Medicine
Stem cell technology holds great potential for improving the sight of people with a visual impairment, particularly to replace photoreceptors that have been lost due to degeneration. A number of studies are currently being undertaken in order to develop new therapies to treat, or prevent a loss of vision. Central to this research is the development of our understanding of how different types of stem cells behave, and how best to harness their potential in the eye. It is important to realise that stem cells are not a one-stop, generic cure, but they do hold exciting potential for vision repair. Read more about cell therapy and regenerative medicine here.
Retina Implant Technology
Retina implant technology involves the use of microelectronics and microchip electrodes surgically implanted into the back of the eye (retina) to restore the function of the damaged light-activated cells found there. These photoreceptor cells respond to light and convert it to an electrical signal which is passed to nerve cells in the eye, and then ultimately to the brain where it is perceived as vision. Click here for more information about retinal implant technology.
Novel Drug Therapy
Sustained scientific research has led to a better understanding of the underlying cause of disease for a large number of retinal degenerative conditions. These efforts have led to the exploration of novel drug therapies to “compensate” for the defective part of how vision is processed. Another approach involves using drugs to target pathways that may slow the death of photoreceptor cells, thus preserving vision for longer. Many of these drugs are repurposed drugs; they may already be approved for use in a completely different disease and are being tested for their effectiveness in the eye. These therapies are attractive as they may have the potential to be administered to many different genetic subtypes independent of the causative gene for the degeneration. Learn more about novel drug therapy here.
Population studies, also known as epidemiology, is the study of a group of individuals taken from the general population who share a common characteristic, such as a health condition. Our Target 5000 research project is a good example of this type of study. Click here for more information about epidemiology.