Therapies under research

Gene therapy works by replacing the defective mutated gene in the affected cells with a normal healthy copy, primarily in inherited retinal disorders.

Gene editing is a technology that enables scientists to precisely alter human DNA to treat genetic diseases or to study disease mechanisms in more detail by re-creating the genetic mutation of interest in a cell model.

Nonsense suppression therapy is a drug-based treatment targeting conditions caused by nonsense mutations. A nonsense mutation introduces an abnormal “stop” signal into a gene that prematurely halts protein production, resulting in a shortened protein that does not function properly.

RNA is involved in protein production from DNA in human cells. Mutations in DNA can be passed down to RNA, and a non-functional protein might be produced. RNA therapies target these "mistakes" in the RNA so that a normal functional protein can be produced instead.

Neuroprotective agents are a broad range of therapies that aim to slow or halt cell degeneration by promoting cell survival and preserving their function.

Optogenetics is technique whereby retinal cells that usually do not have light-detection ability are able to detect light and restore some level of vision. This is achieved by delivering genes coding for the light-detecting protein, opsin to these cells.

Stem cells are immature cells with the ability to develop into different specialised cell types in the body. Apart from being utilised in the laboratory to study diseases and to discover new drugs, it is also being explored as a treatment option for various eye conditions in the form of stem cell transplantation.