It's heartening to share that our lab is actively engaged in developing treatment solutions for children who have mutations in SLC6A1 and GABAA receptors. Here's a detailed overview of our efforts:
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Repurposing 4-Phenylbutyrate (Clinical Trial XX): We are repurposing the drug 4-Phenylbutyrate and conducting a clinical trial named Clinical Trial XX. This approach involves exploring the potential of 4-Phenylbutyrate as a treatment for conditions associated with mutations in SLC6A1 and GABAA receptors. Clinical trials are pivotal in assessing the safety and efficacy of such treatments.
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Exploring Gene Therapy: Gene therapy is an exciting avenue we are exploring. This approach entails introducing functional genes or modifying existing ones to address genetic mutations. We are researching gene therapy as a potential treatment for disorders related to SLC6A1 and GABAA receptors.
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Investigating Antisense Oligonucleotides: Another avenue of exploration involves antisense oligonucleotides (ASOs). These synthetic molecules are designed to target specific RNA sequences, potentially influencing gene expression and protein production. ASOs have demonstrated promise in treating certain genetic disorders by mitigating the effects of genetic mutations.
