Breakthrough in bid to cure blindness as scientists identify gene linked to inherited disease

Posted: Published on July 11th, 2014

This post was added by Dr. Richardson

Advances in plan for individual gene therapies for leading cause of blindness Scientists use stem cells to investigate causes of degenerative eye disease Two patients with retinitis pigmentosa had mutations in a certain gene New gene therapy was used to rescue vision of mice with the same condition

By Julian Robinson

Published: 06:49 EST, 11 July 2014 | Updated: 07:16 EST, 11 July 2014

Scientists have moved closer to a breakthrough in 'personalised' treatments for a leading cause of blindness.

Researchers have stepped up their bid to create individual gene therapies for one of the factors that triggers inherited vision loss.

They used 'induced' stem cells - taken from ordinary skin cells - to investigate patient-specific causes of the degenerative eye disease retinitis pigmentosa (RP), which leads to blindness or severe visual impairment.

Scientists have moved closer to a breakthrough in 'personalised' treatments for a leading cause of blindness

By testing retinal cells created from the stem cells in the laboratory, they linked RP in two patients to mutations in a gene called MFRP.

The scientists then used a virus to deliver normal copies of the gene into the retinal cells and restore their function.

The rest is here:
Breakthrough in bid to cure blindness as scientists identify gene linked to inherited disease

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