Cell and Gene Therapy for Neurodevelopmental Disorders Conference

通过注入工程病毒载体或基因修饰细胞进行基因治疗有可能

Gene therapy via infusion of engineered viral vector or gene-modified cells has the potential to permanently change the health of a patient, potentially sparing them from a lifetime of battling their disease. Although the field of gene therapy is not new, a robust expansion of efforts stoked by the successes of immunotherapy and commercialization of the first products is now occurring. The 2021 conference “Cell and Gene Therapy for Neurodevelopmental Disorders” is an early discussion of how to develop safe and effective treatments for children suffering from neurodevelopmental disorders caused by single – gene defects. New advances in this field are actively studied by the expert speakers for this conference, who are all working on cutting-edge research that has the goal of providing future treatments. The conference is strongly focused on evidence-based science, and an ethics speaker will inform attendees about the risks of false promises from the unregulated “stem cell clinics”. This R13 application seeks funding to support travel awards for graduate or medical students and postdoctoral fellows to attend the conference. At least half of the awards will be reserved for students from traditionally under- represented minority groups. To facilitate trainee interaction with the keynote speakers, the conference will be limited to 150 participants and will feature a trainee/speaker dinner and a trainee lunch workshop, short talks from the travel award winners, and ample opportunity for networking. We are also offering an affordable “virtual attendance” option to broaden the audience. There is a session on “Diversity and International Collaboration” and a panel on “Parent and Patient Advocacy,” with diverse speakers. In bringing novel cell and gene therapy trials from bench to bedside and into routine clinical practice, many scientists, MDs, healthcare, regulatory, manufacturing and other staff members work together in large teams, in consultation with parents and patient advocates. Developing this integrated teamwork to enhance therapy for neurodevelopmental disorders is a central theme of the conference.