Gene therapy against epilepsy
Gene modification defeated seizures
The use of gene therapy made it possible to completely stop seizures of epilepsyYana Aznaurova, "Newspaper.
This method can be a breakthrough in the treatment of the disease suffered by Fyodor Dostoevsky and Vincent van Gogh. In the last 20-30 years, medications have not given epilepsy patients anything other than reducing side effects.
Epilepsy is a chronic disease characterized by convulsive seizures, loss of consciousness and sometimes accompanied by personality changes. The causes of epileptic seizures are still insufficiently studied. It is known that there are two predisposing factors: heredity and organic brain damage. Seizures occur due to uncontrolled excitation of brain neurons and are treated with medications that reduce the excitability of nerve cells.
Officially, 65 million people on Earth suffer from epilepsy. It is believed that several percent of the population has suffered at least one epileptic seizure. Epilepsy can manifest itself in the dream-like states described in 1931 by the British neurologist John Jackson, in which patients experience mainly a variety of sensations of derealization and depersonalization. Other manifestations of epilepsy are speech disorders and psychomotor seizures. Also, epilepsy can manifest itself in seizures, in which there may be no visible convulsions, but only rumbling of the abdomen, diarrhea, or, for example, short-term cardiac arrhythmias. In 1961, a group of Romanian doctors described stroke patients whose epilepsy was expressed only by sweating of the face and head on the side of paralysis.
Epilepsy is often resistant to the action of drugs, and in this case it is necessary to remove the diseased part of the brain. But even this does not guarantee a cure: the effectiveness of the method ranges from 50% to 80%. Therefore, many patients remain without effective treatment.
The method developed by the staff of the Institute of Neurology at University College London is especially important for such patients. Scientists have proposed a potentially effective gene therapy for focal (that is, one in which epileptic discharges originate from one specific part of the brain) epilepsy on a rat model in which epilepsy was caused by injection of tetanus toxin into the motor cortex. The results of their study were published in the journal Science Translational Medicine (Wykes et al., Optogenetic and Potassium Channel Gene Therapy in a Rodent Model of Focal Neocortical Epilepsy).
The scientists used a genetically modified lentivirus labeled with the fluorescent EYFP protein. Lentiviruses are capable of delivering a significant amount of genetic material to the host cell and have a unique ability to replicate in non-dividing cells, which makes lentiviruses convenient for delivering genetic material in research at the molecular level. In the described work, the lentivirus was introduced into epileptic neurons, after which the genome of the virus was embedded in the genome of the neurons themselves. This leads to the fact that the so-called halorodopsin, characteristic of lentiviruses, begins to be produced in "epileptic" neurons. Halorodopsin is a kind of ion pump that increases the potassium content inside the neuron and reduces its excitability.
Gene therapy in a rat model was used to quickly suppress seizures, prevent their recurrence and for the successful treatment of epileptic foci.
"For the first time, gene therapy has been used to completely stop seizures," said the head of the study, Professor Robert Wykes, in an interview with the BBC. – We are hopeful about this method. For the last 20-30 years, medications have not given anything to patients with epilepsy, except to reduce side effects."
Experiments on rats are not enough to use this method. Additional studies will be required, which may take several years before this method can be used in the treatment of patients.
Philip Lee, head of the British charity Epilepsy Action, commented on the results of the study: "It is still very early to talk about the enormous benefits of this method for patients with epilepsy, but we still hope that this will bring us closer to the discovery of a more effective treatment"
Portal "Eternal youth" http://vechnayamolodost.ru26.11.2012