A team of scientists at Iowa University working in conjunction with scientists at Columbia University Medical Centre have succeeded in correcting a genetic mutation causing blindness, in patients’stem cells. Their results were published on 27 January in the Scientific Reports journal. Is curing blindness within our grasp?
For the time being, the team has proved the concept – the first stage before embarking on a clinical trial. Scientists used patients’ reprogrammed skin cells. They repaired the damaged gene in these iPS cells using the CRISPR-Cas9 tool[1]. In theory, these cells can be transplanted into the retina of blind patients. Since the protocol stipulates that a patient’s own cells should be used, the risk of rejection is non-existent and immunosuppressant therapy is not required.
Retina diseases are a perfect model for cell therapies because the surgical techniques employed for the accurate implantation of these cells has already been perfected, explained Professor Mahajan, Professor of Ophthalmology. Nevertheless, although there have been no setbacks, scientists remain cautious about using CRISPR, which could trigger random genome alterations.
[1] Genetic Engineering technique which allows genes to be removed or inserted into cell DNA with the utmost precision.
University of Iowa (27/01/2016)