“There has been a great deal of progress in recent years in developing gene delivery approaches for the treatment of inherited blinding diseases,” Dr. Jean Bennett of the University of Pennsylvania told Digital Trends.
In a research project, published in the journal Human Gene Therapy, a team led by Bennett tested new methods for delivering therapeutic genes directly to the eye. These included two so-called “AAV vectors” named AAV7m8 and AAV8BP2, which have been especially engineered to target specific types of retinal cells. AAV vectors work by transmitting corrected genes and small nucleic acid to individual cells, where they can replicate to reverse the course of rare genetic diseases.
“Because of the promise of these [particular] vectors, we carried out a thorough analysis of the properties of these reagents when delivered to the retina of a large animal,” Bennett continued.
The team tested the therapies on nonhuman primates because only primates possess the macula and fovea parts of the eye which the vectors are designed to work on. In both cases, the vectors were found to work effectively.
The work is all part of an exciting precision medicine program launched by the University of Pennsylvania’s Perelman School of Medicine earlier this year — dedicated to accelerating progress in gene therapies and genome editing to solve a variety of medical conditions.
Next up with this particular piece of research? “The novel AAVs that have been described will be tested for safety and efficacy in particular blinding diseases, first in large animal models and ultimately in human clinical trials,” Bennett noted.
We’ll keep our eyes peeled for future developments!
