In a phase I clinical trial, researchers used gene therapy to cure a genetic disorder causing legal blindness

Those of us dealing in the field of biomed are probably tired of hearing people ask about genetic engineering, and when-oh-when will we be able to cure all the illnesses of the world with it. While genetic engineering does have plenty of potential for treatment of genetic disorders, most of the scientific research in the field screeched to a halt eight years ago, when certain genetically engineered viruses were found to raise the probability of cancer in treated children. And when a field of research is left wide open, someone will usually leap into it.

In the research published today in The Lancet, the authors report of a phase I clinical trial, using gene therapy to cure Leber’s Congenital Amaurosis (LCA) in children and adults. LCA is a rare inherited eye disease, usually characterized by the abnormal development of photoreceptors in the eye. The research team applied a vector – an adeno-virus carrying the normal version of one of the genes that is mutated in some forms of LCA. Each of the participants in the trial was given a single shot in one of his eyes, containing different concentrations of the viruses.

The results were spectacular: the new genes in the cells could now produce the proteins needed to make light receptors work in their retinas. Walking along a dimly lit, simulated street route, the children were able to negotiate barriers they bumped into before the surgery. Another child, who since birth, could only see light and shadows, stared into his father’s face and said he could see the color of his eyes. Later they played soccer together.

Obviously, there is still plenty of work that needs to be done to ensure the safety of gene therapy treatments. All the same, this is a powerful testimony to the promise this field holds.

The Original Article in The Lancet

Press Release by Penn Medicine

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