A gene therapy that restores vision
Washington: In a finding that could lead to
treatment of genetic blindness, American researchers have
developed a gene therapy for an inherited retinal disease that
leads to eventual blindness.
In a research, published in the FASEB Journal, the
scientists at the University of Oklahoma Health Sciences
Center used a non-viral, synthetic nanoparticle carrier to
improve and save the sight of mice with retinitis pigmentosa. "We hope the results of our study will be instrumental
in generating a cure for the debilitating blindness associated
with retinitis pigmentosa and other inherited and acquired
retinal diseases," said senior author Muna I Naash.
"Compacted DNA nanoparticles are an exciting treatment
strategy for these diseases and we look forward to exciting
new developments," Naash said.
In the study, the team treated a group of mice with the
retinal degeneration slow (Rds) gene, which causes retinitis
pigmentosa. The mice received one of 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 analysed by
comparing them to untreated mice with retinitis pigmentosa and
healthy mice with the normal Rds gene.
They also measured the level and pattern of Rds gene
expression, as well as functional, structural and biochemical
improvements in disease symptoms and found that the mice
receiving the nanoparticle gene therapy showed 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.
According to the National Institutes of Health Office
of Rare Diseases Research, retinitis pigmentosa is a group of
inherited eye diseases that affect the retina.
Retinitis pigmentosa causes cells in the retina to die
prematurely, eventually leading to vision loss. There is no
cure for it till now.