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Cell and Gene Therapy for Neurodevelopmental Disorders Conference

神经发育障碍细胞和基因治疗会议

基本信息

批准号：
10237084
负责人：
RANDI J. HAGERMAN
金额：
$ 1万
依托单位：
UNIVERSITY OF CALIFORNIA AT DAVIS
依托单位国家：
美国
项目类别：
财政年份：
2021
资助国家：
美国
起止时间：
2021-09-15 至 2022-08-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/10237084
关键词：
Address Advertising Advocate Archives Award California Cell Therapy Cells Certification Child Clinic Clinical Collaborations Consultations Development Disease Dominant-Negative Mutation Educational workshop Engineering Ethics Face Family Fostering Funding Future Gene Mutation Gene therapy trial Gene-Modified Genes Genetic Goals Grant Health Healthcare Immunotherapy Infusion procedures Institutes International Learning Medical Students Mentors Minority Groups Modification Neuraxis Neurodevelopmental Disorder Nigeria Organ Parents Participant Patient advocacy Patients Persons Postdoctoral Fellow Research Research Personnel Risk Science Scientist Single-Gene Defect Students Talents Travel Treatment Cost Underrepresented Minority Underrepresented Populations Universities Viral Genes Viral Vector Work adeno-associated viral vector base bench to bedside clinical practice clinical translation commercialization developmental disease effective therapy evidence base gene therapy graduate student improved life time cost meetings member new technology novel pediatrician stem cell gene therapy stem cells success symposium targeted treatment underrepresented minority student virtual virtual platform web site

项目摘要

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.

通过输注工程化病毒载体或基因修饰细胞的基因治疗具有潜力，永久改变病人的健康，可能使他们免于一生的战斗，他们的疾病。尽管基因治疗领域并不新鲜，但人们的努力却在不断扩大，通过免疫疗法的成功和第一批产品的商业化，正在发生。2021年会议“神经发育障碍的细胞和基因治疗”是早期讨论如何为患有糖尿病的儿童开发安全有效的治疗方法，由单基因缺陷引起的神经发育障碍。该领域的新进展是这次会议的专家发言人积极研究，他们都致力于尖端技术，旨在提供未来治疗方法的研究。会议重点关注以证据为基础的科学，道德发言人将告知与会者虚假的风险，不受监管的“干细胞诊所”的承诺。此R13应用程序寻求资金支持研究生或医科学生和博士后研究员参加会议至少有一半的奖项将保留给学生从传统的下- 代表少数群体。为了促进学员与主讲人的互动，会议将限于150名与会者，并将举行培训生/演讲者晚宴和学员午餐研讨会，旅游奖获奖者的简短演讲，以及充足的机会，联网我们还提供了一个负担得起的“虚拟出席”的选择，以扩大观众有一个关于“多样性和国际合作”的会议和一个关于 “家长和病人的倡导，”与不同的发言人。将新的细胞和基因疗法从实验室到床边，再到常规临床实践，许多科学家，医学博士，医疗保健，监管、制造和其他工作人员组成大型团队，家长和病人的支持者。发展这种综合团队合作，以加强治疗，神经发育障碍是会议的中心主题。