01.02.2017

Researches Test Efficacy of Gene Therapy For Blindness on Mice and Non-Human Primates

Science's understanding of genetics is growing by the day. Today, many geneticists want to use their knowledge to create gene therapy-based treatments for patients with a genetic predisposition to blindness.

As most people are probably aware, scientists can't simply test a drug on human subjects right off the bat. Before moving to human clinical trials, the drug must first be studied in mice trials and, in some cases, on non-human primates.

This all begs the question: how reliable are the results from the mice and non-human primate trials on average? That's the question put to the test by a group of American researchers at the University of Pennsylvania . These researchers studied the results of gene therapy targeting blindness on mice and non-human primates versus the results obtained from humans.

Researchers involved in this study injected two different strands of adeno-associated virus (AAV) vectors into the mice and non-human primates. These AAV vectors are designed to specifically treat the genes in a person's eyes.

When comparing the results from the mice to human trials, doctors discovered that there's a great degree of difference between the two in terms of safety and efficacy. However, tests in non-human primates were found to be far more accurate and predictive when compared with human trials.

The much-lauded Professor Jean Bennett was at the helm of this study. Professor Bennett works at the University of Pennsylvania's Perelman School of Medicine in the Ophthalmology Department.

People in the medical community are congratulating Dr. Bennett on her latest study. Professor Terence R. Flotte of the University of Massachusetts Medical School told reporters Dr. Bennett's work will, "greatly improve the technology for ocular gene therapy."

The full results of this study were published in a January 2017 edition of the peer-reviewed journal Human Gene Therapy. This particular study was entitled "Evaluation of Dose and Safety of AAV7m8 and AAV8BP2 in the Non-human Primate Retina."


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