27.01.2017

New Study Shows Gene Therapy Might Be Able To Cure Serious Eye Diseases

Laser eye surgery has been used for years to treat people with near- or far-sightedness. Although laser eye surgical procedures such as LASIK have had high success rates with these cases, people with more serious eye problems often fail to receive any benefit from laser treatment.

However, all hope is not lost for people suffering from complicated congenital eye issues. New studies are being conducted every day looking into the efficacy of gene therapy for eye treatment.

The latest study into gene therapy's potential to cure serious eye conditions comes out of the University of Pennsylvania. The team of researchers here specifically looked into two "AAV vectors" which are officially called AAV8P2 and AAV7m8. Geneticists often work with AAV vectors, which are molecules that have the ability to modify and correct genes within the body.

The particular AAV vectors scientists worked with in this study are specifically designed to change the cell structure within the eye's macula and fovea. Both the macula and the fovea are in the eye's retina.

In order to measure the efficacy of these vectors, the researchers had to inject them into nonhuman primates that have both the macula and fovea in each eye.

The tests using these AAV vectors were very promising for members of the ophthalmological community. Eye care specialists believe gene therapy may be the way of the future for restoring sight to those born with a serious congenital eye disease.

The next step for researchers is to test whether or not these AAV vectors can actually cure a patient who is already blind. To figure this out, the professors involved in this study are planning on injecting these AAV vectors into primates with serious eye diseases. If these tests prove successful, they will move on to human clinical trials.

These gene therapy trials are a part of the University of Pennsylvania's Perelman School of Medicine's precision medicine program. This program is focused on using gene therapy and genome editing to cure many rare genetic disorders.

The head researcher on this study was Dr. Jean Bennett, a professor of Ophthalmology at the Perelman School of Medicine. Dr. Bennett told reporters she is confident about the future of gene delivery approaches to treating many serious inherited eye diseases.

For those interested in reading more about this study, the entire report was published in the October 2016 issue of Human Gene Therapy . The study was listed under the title "Evaluation of Dose and Safety of AAV7m8 and AAV8BP2 in the Non-Human Primate Retina."


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