Activity-Dependent Gene Therapy Shows Promise to Treat Epilepsy and Brain Circuit Disorders
Neuropsychiatric and neurodevelopmental disorders that affect the central nervous system (CNS), such as epilepsy, can be treated with the gene therapy approach. In order to reduce neuronal excitability, AAV-based (Adeno-associated virus) gene therapy harnesses the biochemical signals that are increased in response to seizure-related activity in an effort to reduce neuronal excitability. By doing this, the treatment was able to produce a persistent anti-epileptic effect that did not interfere with normal behavior in any way. A study published in the journal Science entitled “On-demand cell-autonomous gene therapy for brain circuit disorders” outlines the findings of this study. Scientists from University College London (UCL) have developed an activity-dependent gene therapy system that follows the dynamics of a network in order to treat diseases.
The purpose of this method is to tag neurons in response to electrical activity by using the promoters of immediate early genes (IEGs). The expression of optogenetic tools has previously been driven by IEG promoters in order to manipulate memory by causing the expression of optogenetic tools. The approach is specific for neurons that are involved in pathological network activity, but it is time-limited in the sense that transgene expression can only persist for as long as neurons are hyperactive and participating in pathological network activity. According to researchers, this should work in principle in the human brain, even though it is very different from a normal brain in terms of structure and functioning. The most critical question to answer is how viral vectors are able to reach the right neurons in the brain and how quickly they can do so.
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