Johns Hopkins Scientists Show New Gene Therapy for Vision Loss Safe for Humans

 Johns Hopkins Scientists Show New Gene Therapy for Vision Loss Safe for Humans

A small and preliminary clinical trial conducted by Johns Hopkins scientists has shown an experimental gene therapy that uses viruses to introduce a therapeutic gene into the eye is safe and that it may be effective in preserving the vision of people with wet age-related macular degeneration (AMD).

According to researchers, the therapy reportedly uses a virus — similar to the common cold that has been altered in the lab so that it is unable to cause disease — as the gene carrier, which is injected into the eye. The virus reportedly penetrates retinal cells and deposits the gene, which turns the cells into factories for the production of a therapeutic protein called sFLT01. Turning the retinal cells into "factories", says researchers, eliminates the need for regular injections of proteins to bind and inactivate vascular endothelial growth factor (VEGF) in the retina.

Researchers believe their study is a promising step towards a new approach that will not only reduce doctor visits and the anxiety and discomfort associated with repeated eye injections, but may also improve long-term outcomes through prolonged VEGF suppression.

The study was recently published in the journal The Lancet.

Click here to read the full press release.

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Source: Johns Hopkins Medicine

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