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▎WuXi AppTec content team editor
Epilepsy is a brain disorder caused by overactive neurons, and about 30% of people with epilepsy do not benefit
from medication.
Recently, a study published in Science proposed a gene therapy that precisely targets abnormally active neurons, which can help abnormal neurons return to normal, thereby providing new therapies
for epilepsy and other brain diseases.
to patients around the world.
What do you know about these innovative therapies? At the end of the article, we have prepared a questionnaire for innovative technology and therapy awareness, welcome to scan the QR code at the end of the article to participate
.
At this moment, every subtle movement of your hand, trying to decipher this line, and even the potential change of mood is inseparable from the sincere cooperation of brain neurons, each of which will control its own activity and electrical signal strength, and accurately transmit information to other cells to ensure normal human behavior
.
But for some people with brain disorders, especially those with epilepsy, their neurons are sometimes overactive, which causes a small percentage of cells to fire abnormally, causing intermittent tics and confusion
.
Although there are medications that can reduce the frequency and severity of seizures, about 30% of people with epilepsy do not benefit
from medications.
Image source: 123RF
Scientists have considered using gene therapy to control overactive neurons, but the difficulty is how to isolate these abnormal neurons without affecting the healthy neurons
on the side.
A recent study in Science made a major breakthrough in addressing this problem, and co-corresponding author Dr.
Gabriele Lignani said: "We have developed gene therapies that can precisely target abnormally active neurons and restore their activity to normal
.
”
To find abnormal neurons, you must first obtain a special genetic marker
for seizures.
Using fluorescent labeling, the authors found that a gene called c-FOS "lights up"
rhythmically under the microscope following epilepsy.
C-FOS is actually a class of early genes, that is, they are briefly and rapidly activated
when exposed to various stimuli.
The promoter of the c-FOS gene, which also responds rapidly to increased neural activity, has also been used to identify cells
that are overactive during epilepsy.
Now that they have genetic indicators, researchers are beginning to think about how to "cool down"
these cells.
If the c-FOS promoter can be followed by genes that reduce cell activity, the system can work
specifically in overactive neurons.
In the authors' analysis, potassium ion channels appear to be a good choice, and this ion channel plays an important role
in regulating neuronal firing.
They selected the KCNA1 gene, which is associated with reduced cell activity, as a partner for the c-FOS promoter, and constructed a gene therapy molecule with the help of adenovirus vectors, so that only those cells whose activity is "overheated" and express c-Fos will be treated
specially.
In mouse models, the authors tried injecting mice with a constructed gene therapy vector, or a control vector, into the hippocampus, and after two weeks of treatment, they used a special drug to cause epilepsy in mice
.
Compared with the control group, mice treated with gene therapy had significantly lower seizure frequency and fewer abnormally active cells
.
In addition, it was more difficult for these mice to reach the action potential threshold at the time of epilepsy.
The authors also tried to obtain a number of brain-like tissue structures with the help of human stem cells, and they also achieved similar positive results
in these tissues.
According to their observations, this gene therapy does not affect the cognitive behavior of mice when it inhibits epilepsy, which is safe
.
Compared to past drug tests in the same model, gene therapy reduced spontaneous epilepsy
by 80%.
The researchers point out that this new gene therapy concept could theoretically be used to target other neurological diseases, such as Parkinson's disease
.
"Our study means that abnormal brain cells can be restored to normal, and this approach can be used to intervene in neurological diseases
that are not sensitive to drugs.
" Study co-corresponding author Professor Dimitri Kullmann said
.
The biomedical industry is entering a new era
.
In addition to traditional chemical and biological drugs, a series of new types of therapies have gradually come to the fore and become a hot spot in recent years, accelerating the breakthrough
of patients around the world.
In order to understand the familiarity of universities and scientific research institutions with innovative technologies and therapies, you are cordially invited to scan the QR code below or click to read the original article and spend 1 minute filling out the questionnaire
.
Based on the survey results, your concerns will have the opportunity to be answered
at WuXi AppTec's New Year virtual event.
We will randomly select 15 students who participated in the questionnaire to give gifts
.
References:[1] Yichen Qiu et al, On-demand cell-autonomous gene therapy for brain circuit disorders, Science (2022).
DOI: 10.
1126/science.
abq6656.
[2] Gene therapy targeting overactive brain cells could treat neurological disorders.
Retrieved November 10, 2022 from https://medicalxpress.
com/news/2022-11-gene-therapy-overactive-brain-cells.
html
