Researchers have engineered a new adeno-associated virus that could greatly expand gene therapy to help restore sight to patients with blinding diseases like retinitis pigmentosa and macular degeneration.
This diagram shows an adeno-associated virus. Prof Schaffer and colleagues changed ten amino acids in one of its coat proteins, shown in orange, to get it to pass through retinal cells to the target photoreceptors.
Over the last six years, several teams of scientists have successfully treated people with a rare inherited eye disease by injecting a virus with a normal gene directly into the retina of an eye with a defective gene. Despite the invasive process, the virus with the normal gene was not capable of reaching all the retinal cells that need fixing.
“Sticking a needle through the retina and injecting the engineered virus behind the retina is a risky surgical procedure,” explained Prof David Schaffer of the University of California, Berkeley, senior author of a paper published in the journal Science Translational Medicine.
“But doctors have no choice because none of the gene delivery viruses can travel all the way through the back of the eye to reach the photoreceptors – the light sensitive cells that need the therapeutic gene.”
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