There are over 285 million visually impaired people in the world, of whom 39 million are blind and 246 million have moderate to severe visual impairment, suggests the World Health Organization.
Many gene therapy-based approaches are in development to combat
genetic and other causes of blindness and vision loss. Much can be
learned about the safety and effectiveness of these promising new
therapies by studying them first in non-human primates before initiating
clinical trials, suggest the results of a study published in Human Gene Therapy
‘The safety and effectiveness of ocular gene therapy-based approaches can be studied first in non-human primates before initiating clinical trials.’
A team of researchers led by Jean Bennett, Scheie Eye Institute,
University of Pennsylvania, Perelman School of Medicine, Philadelphia,
evaluated and compared two adeno-associated virus (AAV) vectors designed
to deliver therapeutic genes directly to the eye when administered via
subretinal injection to non-human primates.
They report the differences
in gene expression efficiency and immunogenicity across different doses
in the article entitled "Evaluation of Dose and Safety of AAV7m8 and AAV8BP2 in the Non-human Primate Retina."
Gene therapies developed and tested in mice may present quite
different safety and efficacy profiles when administered to humans.
Evaluating gene delivery vectors in non-human primates provides results
that are more predictive of what one can expect in humans.
"Dr. Bennett and her team have already made major breakthroughs in
gene therapy for genetic causes of blindness. The work published here
promises to greatly improve the technology for ocular gene therapy,"
says Editor-in-Chief Terence R. Flotte, Celia and Isaac Haidak
Professor of Medical Education and Dean, Provost, and Executive Deputy
Chancellor, University of Massachusetts Medical School, Worcester, MA.