“Gene therapies are an exciting area of biotechnology and clinical therapies right now. Our team in Sydney is focused on developing gene therapy for individuals with variants in the VAMP2 gene where we are connecting with many research teams and clinicians worldwide to assist us in our quest. In support of this universal approach to our research, we have adapted the famous African proverb to: It takes a village to raise a child but an army of researchers, clinicians, and communities to cure one”.
“What you have done has been really helpful. I would like to see that done for all the rare genes that we work on. It is helpful for families to meet and chat and compare the good and bad as a patient support group. Ideally this would encourage further research and understanding of rare diseases".
"I am touched by your email and active efforts. There really isn't much more about VAMP2 I can tell you. You are aware of everything that is going on. Let's keep in touch as things progress. I am happy to help support you in what you are doing. The world needs more parents like you".
“I am not a medical doctor. I am studying neurotransmitter release - a mechanism by which neurons communicate with each other in the brain. VAMP2 is a major player in this process. It was your website that motivated me to start researching disorders caused by VAMP2 mutations. Your website is very detailed, and I could see how this could help us better understand what is happening with neuronal signaling when VAMP2 malfunctions”.
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