Retinal Degeneration May Be Preventable with New Gene Therapy

A study recently published by the Tufts University School of Medicine, along with the Sackler School of Graduate Biomedical Sciences at Tufts, has shown that gene therapy has the ability to delay the development of some eye diseases that destroy vision. Nanoparticles were used to send therapeutic genes directly to the retina. Mice receiving the treatment retained more of their eyesight temporarily than the untreated mice.

This research is groundbreaking in it’s use of nanoparticles instead of viral gene delivery. Nanoparticles can penetrate cells without damaging or disrupting them. They also don’t trigger the immune system like more traditional forms of gene therapy, where a deactivated virus is used to deliver new DNA to certain cells.

Researchers used the nanoparticle gene therapy methods to deliver a gene to produce Glial Cell Line-Derived Neurotrophic Factor. This protein has been shown to protect key photoreceptor cells that are crucial to vision. The retinas of mice treated experienced much lower cell losses than those of the control group. By keeping these photoreceptor cells alive, these mice retained much more of their eyesight after seven days.

Unfortunately, the eyesight preservation caused by this treatment wore off after fourteen days, with both groups of mice experiencing the same loss of eyesight. This means the treatment is still only a temporary procedure.

Researchers hold to prolong the treatment by combining elements into the DNA strands that let it replicate and remain in the retina cells. If they can do so, many other diseases and conditions may be able to be treated with this new form of non-viral gene therapy.

Previous research done by the same team that worked on this study discovered the non-viral delivery method used. Researchers found that a special peptide, known as PEG-POD, can compact the desired DNA strands into nanoparticles. This breakthrough allows for an easier delivery process and less disruption to the cells.


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