Biology and Biotechnology of Cell and Gene Therapy

细胞和基因治疗生物学和生物技术

• 批准号: 10090424
• 负责人: DAVID V SCHAFFER
• 金额: $ 35.65万
• 依托单位: UNIVERSITY OF CALIFORNIA BERKELEY
• 依托单位国家: 美国
• 财政年份: 2021
• 资助国家: 美国
• 起止时间: 2021-07-01 至 2026-06-30
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10090424
• 关键词: Biology; Biotechnology; Cell Therapy; gene therapy

Project Summary Graduate education has traditionally been successful in educating students in either engineering or the biomedical sciences, but the disparate nature of the scientific and engineering backgrounds necessary to successfully move the gene and cell therapy field forward requires novel educational approaches and methods that integrate these disciplines. With small molecule and protein therapies well-established in the pharmaceutical and biotechnology industries, gene and cell therapy represent the next generation of therapeutics to address serious unmet medical need. Because these therapeutics involve the delivery of DNA – in the form of genes or entire genomes – they have the potential to provide long-term therapeutic benefit following a single administration. However, the gene and cell therapy field face complex biological and technological challenges. Delivery of genetic constructs, either in vitro or in vivo, must be improved, and in addition therapeutic payloads including CRISPR/Cas9 and other genome editing machinery requires improved potency. In addition, cellular targets including human embryonic stem cells (hESCs), induced pluripotent stem cells (iPSCs), and adult stem cells are difficult to precisely control, since the repertoire of signals and cues that naturally control stem cell self-renewal and differentiation are not well understood, yet precise control is essential to unlocking applications in tissue engineering and regenerative medicine. The University of California at Berkeley has developed the highly interdisciplinary Biology and Biotechnology of Cell and Gene Therapy (BBCGT) Training Program. With the involvement and support of our 24 faculty, the Berkeley Stem Cell Center, Bioengineering Department, Molecular and Cell Biology Department, and Helen Wills Neuroscience Institute, we have designed and have been successfully implementing a program to support the education and training of predoctoral fellows in gene and cell therapy. This newly emerging discipline represents the convergence of the biological and biomedical sciences, physical sciences, engineering, and ethics. The primary objectives of our program have therefore been to formally organize the structure and scope of new training opportunities in this rapidly expanding discipline, to dissolve traditional academic barriers to interdisciplinary graduate science education, and to provide strong research training in academia and industry. As part of these efforts, we will immerse trainees in a Biology and Biotechnology of Cell and Gene Therapy curriculum, training in the responsible conduct of research, seminar series, annual retreat, interdisciplinary research, career development resources, industrial internship experience, and participation in a recruitment and retention plan to enhance diversity. The resulting program will be highly effective in training young scientists to work at the interface of the biomedical sciences and engineering in a rapidly-evolving, impactful, and timely area of biomedical research.

项目摘要 研究生教育在工程或工程学的教育学生或 生物医学科学，但科学和工程背景的不同性质 成功地移动基因和细胞疗法领域需要新颖的教育方法和方法 将这些学科与小分子和蛋白质疗法结合在一起 药物和生物技术行业，基因和细胞疗法治疗疗法下一代 解决严重的未满足医疗需求的治疗方法。 - 基因或整个基因组的形式 - 它们具有有效的能力来提供长期治疗益处 但是，一个给药。 必须改善技术挑战，无论是体外还是体内 补充的治疗有效载荷包括包括CRISPR/CAS9和其他基因组编辑机械需要改进 另外。 细胞（IPSC）和成年干细胞是精确控制的，因为信号和提示的曲目 自然控制的干细胞自我更新和区分尚不清楚，而精确的控制是 解锁组织工程和再生医学的应用 伯克利的加利福尼亚州开发了细胞的高度跨学科生物学和生物技术 基因疗法（BBCGT）培训计划。 干细胞中心，生物连接系，分子和细胞生物学系和海伦·威尔斯 神经科学研究所，我们已经设计并已成功掩盖了一个支持您的计划 基因和细胞疗法中的专用研究员的教育和培训。 代表生物学和生物医学科学，物理科学，工程学的融合 道德规范。因此，我们计划的主要目标是正式组织结构 在这种迅速扩展的学科中，新的培训机会，以使传统的学术障碍变形 跨界研究生科学教育，并在学术界和行业中提供强大的研究培训。 作为主体的一部分，我们将在细胞和基因疗法的生物学和生物技术中进行IMERSE培训 课程，培训在国际研究中的培训，研讨会系列，年度静修，跨学科的培训 研究，职业发展资源，工业实习经验和参与招聘 保留计划以增强多样性。 科学家在生物医学科学与工程的界面中工作 和及时的生物医学研究领域。