Gene Therapy (Somatic)
Research | General Research | Gene Therapy (Somatic)
This is where therapeutic genes are inserted into tissue or cells to produce a naturally occurring protein or substance that is lacking or not functioning correctly in an individual patient.
Inherited retinal diseases are a diverse group of conditions that result from mutations in any one of over 100 different genes. Many of the genes have been identified and more is known about how they function. There is still further work to be done to identify and understand the remaining genes. The existing knowledge, however, gives a major impetus to develop gene-based treatments or therapies. The eye offers many unique advantages as a target organ for the development of gene therapy. It is easily accessible and allows localised exposure of the target tissue to therapeutic agents with limited risk of systemic effects.
Recently there has been a significant step forward in the form of clinical trials to test a treatment for blindness in children caused by Leber's Congental Amaurosis (LCA) by researchers at UCL (University College London) and Moorfields Eye Hospital. This involves the first trials (on adults with the disease) inserting healthy copies of the gene into the cells of the retina to help them to function normally. Read more...(link to page with release about this) Clinical trials are also taking place in centres in other countries, including the U.S.A., into the LCA gene and genes linked to Stargardts disease.
Encapsulated cell therapy (ECT) refers to the insertion of a tiny device containing genetically modified retinal cells, which release a protein called ciliary neurotrophic factor (CNTF), into the eye. The hope is that the protein will "rescue" photoreceptors and reduce vision loss in some patients with different forms of retinal degeneration.