Gene Therapy Using Nanoparticle Delivery Restores Vision in Mice

Filed in archive Diagnostics, Methodologies and Instrumentation , Gene Therapy , Nanomedicine on April 6, 2010

© Kyle May

In a research report published in the April 2010 print issue of The FASEB Journal, scientists describe how they were able to reverse a type of blindness in mice using a form of gene therapy that does not involve the use of modified viruses. They used a non-viral, synthetic nanoparticle carrier to improve and save the sight of mice with retinitis pigmentosa, a genetic disease characterized by progressive vision loss and eventual blindness.

Using mice with the retinal degeneration slow (Rds) gene, which causes retinitis pigmentosa, the mice were given three types of "treatments:" nanoparticles containing the normal copy of the Rds gene, the normal gene alone, or saline solution. After these treatments were delivered to the mice, the structure and function of the retina were analyzed by comparing them to untreated mice with retinitis pigmentosa and healthy mice with the normal Rds gene. Researchers also measured the level and pattern of Rds gene expression, as well as functional, structural and biochemical improvements in disease symptoms.

They discovered that mice receiving the nanoparticle gene therapy show significant signs of healing. These mice had structural improvement in their retinas, as well as functional vision improvements, which lasted throughout the duration of the study. The mice that received the gene alone or saline continued to lose their vision. The nanoparticles were safe and well-tolerated with no adverse effects.