Gene therapy of retinal pigmented dystrophy: genes + nanoparticles
A safe way of delivering genetic information using nanoparticles is shown
Dmitry Safin, Compulenta
American experts have demonstrated a safe way of delivering genetic information using nanoparticles and proved its effectiveness in experiments on mice suffering from retinal pigmented dystrophy.
Pigmented dystrophy refers to hereditary diseases and is characterized by the consistent destruction of rods and cones in the retina (in the picture, the affected areas have a dark color).
As a result, the patient's field of vision narrows, and in many cases everything ends in blindness.
The authors worked with an experimental model of the disease – mice carrying a mutation in the Rds gene encoding the protein peripherin-2 (peripherin 2). On the 5th or 22nd day after birth, the animals of the experimental group were injected with nanoparticles containing the "correct" copy of Rds, and the control groups were injected with plasmid DNA containing the same gene, or simply a saline solution.
It is reported that the content of matrix RNA corresponding to Rds in animals that received nanoparticles was established at a higher level than in mice from the control groups, and remained elevated on the 120th day of the experiment. The functional state of the retinal rods, assessed by the method of electroretinography, improved somewhat, and the destruction of the cones was prevented. At the same time, rodents in all other groups continued to gradually lose their eyesight.
The researchers did not note any side effects in the case of the use of nanoparticles.
The full version of the report is published in The FASEB Journal (Xue Cai et al., Gene delivery to mitotic and postmitotic photoreceptors via compact DNA nanoparticles results in improved phenotype in a mouse model of retinitis pigmentosa).
Prepared based on the materials of the Federation of American Societies of Experimental Biology (Making the blind see: Gene therapy restores vision in mice).
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