Precision Genome Engineering

摘要 要求为题为“精确基因组工程”的Keystone专题讨论会提供支助， 作者：Laura Sepp-Lorenzino和Matthew Porteus博士会议将在加拿大卑诗省惠斯勒举行 2023年3月19日至23日。 基因组编辑是一种强大而精确的工具，可以改变细胞DNA的序列， 阐明疾病机制和开发新的治疗方法，以解决潜在的病理学， 基因组、表观基因组和转录组水平。对基因组编辑如何工作的基本理解， 基因组编辑新工具的开发以及临床应用正在快速发展。 同样，基因编辑正在彻底改变生物学和治疗学。不断扩大的编辑工具箱 酶和递送解决方案使基因组的精确操作成为可能，它正在推动多种多样的 在细胞生物学、植物和动物科学以及人类治疗学中的应用管道。新出现的临床 来自基因编辑细胞疗法的数据沿着来自体内递送的基因编辑药物的临床前数据 候选人正在证明可行性，多功能性，以及在人类中的有效性和安全性的潜力。 此外，人们对作物和牲畜的基因编辑有很大的兴趣和投资。因此本 Keystone研讨会将涵盖基因组编辑领域的关键方面，从基础 从生物化学到工具开发，再到生物学、农业和医学的应用。会议将 学术界和生物制药界的科学家，包括监管机构和伦理学家都感兴趣。而且这 会议还将独特地汇集研究自然基因组编辑系统的科学家， 自然DNA损伤修复与科学家试图通过开发新的基因组编辑系统， 一个会议的并列集中在基因组不稳定性和DNA修复。这些联合会议将 分享两个双主题讲座和两个全体会议：基因组不稳定性和癌症与基因组 编辑和DNA修复与会者还将分享用餐时间和海报，以加强网络 机会此外，该会议计划包括职业发展研讨会，并设有一个 关于基因组编辑伦理的小组讨论。该计划还旨在提供充足的机会 挑选初级参与者参加讲习班演讲以及全体会议期间的简短演讲。

ABSTRACT Support is requested for a Keystone Symposia conference entitled Precision Genome Engineering, organized by Drs. Laura Sepp-Lorenzino and Matthew Porteus. The conference will be held in Whistler, BC, Canada from March 19-23, 2023. Genome editing is a powerful and precise tool to change the sequence of cellular DNA for both better elucidating disease mechanisms and for developing new treatments to address underlying pathologies at the genomic, epigenomic and transcriptomic level. The basic understanding of how genome editing works, the development of new tools in genome editing, and the translation to clinic are evolving at a rapid pace. Likewise, gene editing is revolutionizing biology and therapeutics. An ever-expanding toolbox of editing enzymes and delivery solutions enable the precise manipulation of the genome, and it is fueling a diverse pipeline of applications in cell biology, plant and animal science and human therapeutics. Emerging clinical data from gene edited cell therapies along with preclinical data from in vivo delivered gene editing drug candidates are demonstrating the feasibility, versatility, and potential for efficacy and safety in humans. Furthermore, there is great interest and investment into gene editing for crops and livestock. Therefore, this Keystone Symposia conference will cover key aspects of the genome editing field, from fundamental biochemistry to tool development, to applications in biology, agriculture, and medicine. The conference will be of interest to scientists from both academia and biopharma, including regulators and ethicists. Moreover, this conference will also uniquely bring together scientists who have studied natural genome editing systems and natural DNA damage repair with scientists trying to develop novel genome editing systems through the juxtaposition of a conference focused on Genomic Instability and DNA Repair. These joint conferences will share two dual keynote lectures and two plenary sessions: Genome Instability and Cancer and Genome Editing and DNA Repair. Participants will also share mealtimes and poster sessions to enhance networking opportunities. In addition, this conference program includes a Career Development Workshop and features a panel discussion on Genome Editing Ethics. The program was also designed to provide ample opportunities for junior participates to be selected for workshop presentations as well as short talks during plenary sessions.