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: 10621376
• 负责人: Dirk Hockemeyer
• 金额: $ 39.08万
• 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/10621376
• 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 研究生教育在传统上成功地培养了工程或机械专业的学生。 生物医学科学，但科学和工程背景的不同性质， 成功地推动基因和细胞治疗领域的发展需要新的教育途径和方法 整合这些学科。随着小分子和蛋白质疗法的成熟， 在制药和生物技术行业，基因和细胞治疗代表了下一代的 治疗方法来解决严重未满足的医疗需求。因为这些疗法涉及DNA的输送 - 以基因或整个基因组的形式-它们有可能提供长期的治疗益处 在一次管理之后。然而，基因和细胞治疗领域面临着复杂的生物学和 技术挑战。无论是在体外还是在体内，遗传构建体的递送都必须得到改善， 此外，包括CRISPR/Cas9和其他基因组编辑机制在内的治疗有效载荷需要改进。 力量此外，包括人胚胎干细胞（hESC）、诱导的多能干细胞（iPS）和人胚胎干细胞（hES）在内的细胞靶点也被广泛应用。 细胞（iPSC）和成体干细胞难以精确控制，因为 天然控制干细胞自我更新和分化还没有很好地理解，然而精确的控制 这对解锁组织工程和再生医学的应用至关重要。大学 加州伯克利分校发展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。在我们24名教师的参与和支持下，伯克利 干细胞中心，生物工程系，分子和细胞生物学系，和海伦威尔斯 神经科学研究所，我们已经设计并成功实施了一项计划，以支持 基因和细胞治疗博士前研究员的教育和培训。这门新兴学科 代表了生物和生物医学科学，物理科学，工程学的融合， 伦理因此，我们计划的主要目标是正式组织结构和范围 在这个迅速扩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，我们将让学员沉浸在细胞和基因治疗的生物学和生物技术中。 课程，负责任地进行研究的培训，系列研讨会，年度务虚会，跨学科 研究，职业发展资源，工业实习经验，并参与招聘 和保留计划，以提高多样性。由此产生的计划将是非常有效的培训年轻人 科学家在生物医学科学和工程的接口工作，在一个快速发展，有影响力， 和及时的生物医学研究领域。

项目成果

A Weak Link with Actin Organizes Tight Junctions to Control Epithelial Permeability.

• DOI: 10.1016/j.devcel.2020.07.022
• 发表时间: 2020-09-28
• 期刊: Developmental cell
• 影响因子: 11.8
• 作者: Belardi B;Hamkins-Indik T;Harris AR;Kim J;Xu K;Fletcher DA
• 通讯作者: Fletcher DA

Adeno-associated virus-mediated delivery of CRISPR-Cas9 for genome editing in the central nervous system.

• DOI: 10.1016/j.cobme.2018.08.003
• 发表时间: 2018-09
• 期刊: Current opinion in biomedical engineering
• 影响因子: 3.9
• 作者: Fuentes CM;Schaffer DV
• 通讯作者: Schaffer DV

Optimizing COVID-19 control with asymptomatic surveillance testing in a university environment.

• DOI: 10.1016/j.epidem.2021.100527
• 发表时间: 2021-12
• 期刊: Epidemics
• 影响因子: 3.8
• 作者: Brook CE;Northrup GR;Ehrenberg AJ;IGI SARS-CoV-2 Testing Consortium;Doudna JA;Boots M
• 通讯作者: Boots M

Unconventional secretion of α-synuclein mediated by palmitoylated DNAJC5 oligomers.

由棕榈酰化的DNAJC5低聚物介导的α-突触核蛋白的非常规分泌。

• DOI: 10.7554/elife.85837
• 发表时间: 2023-01-10
• 期刊: ELIFE
• 影响因子: 7.7
• 作者: Wu, Shenjie;Villegas, Nancy C. Hernandez;Sirkis, Daniel W.;Thomas-Wright, Iona;Wade-Martins, Richard;Schekman, Randy;Pfeffer, Suzanne R.
• 通讯作者: Pfeffer, Suzanne R.

Chemical disaggregation of alpha-synuclein fibrils as a therapy for synucleinopathies.

• DOI: 10.1073/pnas.2300965120
• 发表时间: 2023-03-14
• 期刊: PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
• 影响因子: 11.1
• 作者: Wu, Shenjie;Villegas, Nancy C. Hernandez;Schekman, Randy
• 通讯作者: Schekman, Randy