WIKIMEDIA, SHELOVESGHOSTSUsing mouse eyes as a setting for directed evolution, scientists have created a new version of the gene therapy vector adeno-associated virus (AAV) that can deliver genes deep into the retina, according to a paper published online today (June 12) in Science Translational Medicine. Such a vector could improve therapeutic gene delivery to target cells and lead to safer and less invasive gene therapy treatments.
“This is a beautifully planned, executed, and powerfully presented paper,” said Jean Bennett, a professor of ophthalmology at the University of Pennsylvania in Philadelphia, who was not involved in the study. “It shows the results of a very clever system to evolve AAV to target cells in the retina efficiently from an intravitreal injection.”
Intravitreal injection, whereby a needle is pushed into the eye’s vitreous, or gel-like core, is a common drug delivery procedure performed under local anesthetic in a doctor’s office, explained Bennett. But using this routine injection technique in trials of gene therapy for retinal degeneration has thus far proven impossible.
The problem, explained David Schaffer, a professor of chemical and biomolecular engineering, bioengineering, and neuroscience at the University of California, Berkeley, who ...
