A new method of gene therapy for Leber's amaurosis
Gene therapy against blindness
Mednovosti based on the materials of Medicalexpress: Scientists test new gene therapy for vision loss from a mitochondrial disease A group of researchers from the University of Miami Miller School of Medicine has proposed a new way to combat Leber's hereditary optic neuropathy, a serious disease that leads to blindness.
The cause of the disease is a mutation that occurs in the mitochondrial genome. Mitochondria are energy stations of cells, the peculiarity of which is the presence of their own genome. Most often, the ND4 gene is damaged in patients with hereditary Leber optic neuropathy. The disease usually debuts in adolescence and is accompanied by a rapid loss of visual acuity associated with the destruction of retinal neurons.
John Guy and his colleagues first developed a model of the disease in laboratory mice, and only then tested a new treatment method on it. The diseased animals developed optic nerve atrophy, retinal nerve cells were destroyed, and vision rapidly deteriorated.
Using a modified adenoassociated virus, scientists were able to inject a full-fledged copy of the ND4 gene into damaged mitochondria using injections into the eye. This helped to significantly improve the rodents' eyesight. The introduction of the virus to healthy mice did not lead to any side effects.
Scientists note that the results of the study exceeded their expectations – gene therapy was able not only to prevent the development of blindness, but also helped to restore the vision almost completely lost by mice. In the near future, it is planned to conduct clinical trials with the participation of patients suffering from hereditary Leber optic neuropathy.
Article by Yu et al. The consequences of zygote injection and germline transfer of mutant human mitochondrial DNA in mice are published in PNAS – VM.
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06.10.2015