Vincent Lertchareonyong Rose Hills
CRISPR-Mediated Knockout of Epileptic Drug Targets
Epilepsy affects an estimated 50 million worldwide, and despite over 30 FDA-approved anti-epileptic medications, 30–40% of patients are resistant to treatment, highlighting the need for alternative approaches. Rather than focus on small molecule discovery, which is often non-specific and has limited efficacy in epilepsy treatment, this project explores gene therapies that may overcome these barriers. Utilizing state-of-the-art technology and established methods, I plan to investigate gene therapies to modulate expression of key protein targets, advancing the design of therapies that reduce epileptiform activity. I aim to target two therapeutically relevant proteins involved in epileptiform activity—SV2A and GRIK2—using CRISPR-mediated gene knockout. SV2A is crucial for synaptic vesicle trafficking and neurotransmitter release, and its modulation has been linked to reduced seizures. GRIK2, a glutamate receptor subunit, contributes to excitatory signaling; its dysregulation is associated with neuronal hyperexcitability. Disrupting these proteins may reduce aberrant activity and offer more targeted therapies.
Message To Sponsor
Thank you for your generous support of my summer research. This opportunity allows me to dive deeper into my interest in neuroscience and genetics through hands-on work that I’m truly passionate about. Being part of a project with real therapeutic potential is both inspiring and motivating. I’m very grateful to the foundation for making this experience possible.