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 永久性地改变患者的健康，可能使他们免于终生抗争 他们的病。尽管基因治疗领域并不新鲜，但一项强有力的努力推动了 由免疫疗法的成功和商业化的第一批产品现在 发生的。2021年“神经发育障碍的细胞和基因治疗”会议是 早期探讨如何开发安全有效的治疗儿童慢性支气管炎 由单基因缺陷引起的神经发育障碍。这一领域的新进展是 本次会议的专家演讲者积极研究，他们都致力于尖端技术 以提供未来治疗为目标的研究。这次会议的重点是 以证据为基础的科学，一位伦理演讲者将告知与会者虚假的风险 来自不受监管的“干细胞诊所”的承诺。此R13应用程序寻求资金以支持 研究生或医科学生和博士后研究员参加 会议。至少一半的奖项将保留给来自传统年龄以下的学生- 代表少数群体。为方便学员与主讲嘉宾互动， 会议将限制为150人参加，并将包括学员/演讲者晚宴和 学员午餐工作坊，旅游奖获得者的简短演讲，以及充足的机会 网络。我们还提供负担得起的“虚拟出席”选项，以扩大 观众。有一个主题为“多样性和国际合作”的会议和一个关于 “家长和病人的倡导”，有不同的演讲者。在带来新的细胞和基因治疗方面 从长凳到床边再到常规临床实践的试验，许多科学家、医学博士、医疗保健人员、 监管、制造和其他工作人员在大型团队中一起工作，进行咨询 与父母和患者权益倡导者一起。发展这种综合的团队合作，以加强治疗 神经发育障碍是这次会议的一个中心主题。

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.