Potential Theraupatic Application of CRISPR Gene Editing System in Dravet Syndrome

Dravet syndrome (DS), is a childhood-onset severe form of epilepsy which usually is a result of de novo mutations in SNC1A gene that encodes alpha1 Subunit of NaV1.1 (Voltage-gated Na+ channel). Valproic acid as the first line treatment for Dravet syndrome patients only controls seizure which means there is no cure for it up to now. CRISPR as a gene editing tool seems to be an appropriate potential therapeutic strategy for Dravet syndrome. Different CRISPR gene editing systems like base editing and HDR according to type of mutation can be done. Base editing is a potential appropriate strategy for point mutations in which a or T should be changed to G or C or vice versa. HDR is the other system which works via the cells homologous recombination dependent repair system. Delivery of CRISPR component to CNScan be done using AAV vectors which were shown to infect neurons quite efficiently. these vehicles can be administered to patient either systemically, intrathecally or intracranially. This procedure as a personalized medicine can be optimized and fit for every individual needs andalso avoid over treatment and under treatment because some patient still is resistant to the existing treatments In addition to being specific to patients it will be a certain cure and have long lasting effects in contrast to current treatments. Conclusion: Choosing and discovering the best delivery system like AAV based vectors will make this strategy a safe and cost-effective and in brief a typical potential theraupatic strategy for Dravet syndrome.