Stem cell therapy as treatment for Stargardt disease.

Abstract

Stargardt disease or Juvenile Macular Degeneration is a rare genetic disorder caused by a mutation in the ABCA4 gene that results in degeneration of the macula and loss of central vision. The mutation in the ABCA4 gene causes a build-up of lipofuscin, which is a substance that is left over from the breakdown and absorption of damaged blood cells. This build-up of lipofuscin causes the death of photoreceptor cells and the subsequent degeneration of the macula. Due to the nature of Stargardt's disease, there are currently no cures or treatment options. However, in recent years, there has been an increase in research and exploration of utilizing stem cell therapy as a potential cure and treatment for Stargardt disease. Growing research in the field of ophthalmology has found that the use of stem cells can potentially replace the loss of photoreceptor cells, slow the progression of the degeneration of vision, and be a potential new treatment option for Stargardt disease.