Miracle Gene Therapy could cure Rare Blindness


LCA (Leber’s congenital amaurosis) is a rare eye condition which affects children from birth leaving them with severe vision loss. After just one injection with this new gene therapy, children’s eyesight is improving dramatically. Whilst the research of this new development is still in its early stages, it has been successful on all of its tested patients…leaving LCA sufferer’s with growing hope.

LCA is caused by a gene mutation and its effects progress for around 30 to 50 years until they leave the sufferer with complete blindness. The gene therapy then works by replacing this defective gene by injecting the new gene directly into the cell within the eye. The gene is carried within a viral coat as it is inserted into its new cell and once inside the gene then produces healthy enzymes (light sensitive pigments) which are needed to see. 

The advancements of this kind of therapy are staggering and offers hope to 1/80,000 people that LCA affects at birth. The therapy is thought to work best on young children due to more of the cells being salvageable, but has also been successful for adults. The results also offer hope for the whole area of gene therapy and could be developed to help people who suffer from problems such as AMD (age-related macular degeneration). 

The results are being hailed “spectacular” and are nothing short of a miracle as not much hope was given for LCA patients and other eye-disease sufferer’s alike. With early diagnoses of LCA, the majority of the retina should remain with prompt treatment, allowing these children to lead an almost visually clear life.

18th November 2009

<<<more macular degeneration news


Laser Eye Surgery Enquiry Form

Complete this form if you would like information about laser eye surgery including costs & suitablity.