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.

项目概要 传统上，研究生教育成功地教育了工程或应用领域的学生。 生物医学科学，但科学和工程背景的不同性质是必要的 成功推动基因和细胞治疗领域向前发展需要新颖的教育途径和方法 整合了这些学科，并在小分子和蛋白质疗法中得到了广泛应用。 制药和生物技术行业、基因和细胞疗法代表了下一代 解决严重未满足的医疗需求的疗法，因为这些疗法涉及 DNA 的传递。 – 以基因或整个基因组的形式 – 它们有潜力提供长期的治疗益处 然而，单次给药后，基因和细胞治疗领域面临着复杂的生物学和环境问题。 技术挑战。无论是体外还是体内的基因构建体的传递都必须得到改进。 包括 CRISPR/Cas9 和其他基因组编辑机制在内的额外治疗有效负载需要改进 此外，细胞靶标包括人胚胎干细胞（hESC）、诱导多能干细胞。 细胞（iPSC）和成体干细胞很难精确控制，因为信号和线索的全部组成部分 自然控制干细胞的自我更新和分化尚不清楚，但精确控制是 对于解锁组织工程和再生医学的应用至关重要。 加州大学伯克利分校开发了高度跨学科的细胞生物学和生物技术 基因治疗 (BBCGT) 培训计划得到了伯克利分校 24 名教员的参与和支持。 干细胞中心、生物工程系、分子与细胞生物学系、Helen Wills 神经科学研究所，我们设计并成功实施了一项计划来支持 基因和细胞治疗这一新兴学科的博士前研究员的教育和培训。 代表了生物和生物医学科学、物理科学、工程学和 因此，我们计划的主要目标是正式组织结构和范围。 在这个快速发展的学科中提供新的培训机会，以消除传统的学术障碍 跨学科的研究生科学教育，并在学术界和工业界提供强有力的研究培训。 作为这些努力的一部分，我们将使学员沉浸在细胞和基因治疗的生物学和生物技术中 课程、负责任的研究行为培训、研讨会系列、年度务虚会、跨学科 研究、职业发展资源、工业实习经验以及参与招聘 由此产生的计划将在培训年轻人方面非常有效。 科学家们在生物医学科学和工程学的交叉领域开展快速发展、有影响力的工作 以及及时的生物医学研究领域。