Heart Development and Disease: From Genes to Cures

摘要 请求为题为心脏发育和疾病的Keystone研讨会提供支持： 基因治疗，由肖恩·吴博士、沃尔夫拉姆·齐默尔曼博士和克里斯汀·塞德曼博士组织。这次会议 将于2023年2月12日至15日在新墨西哥州圣达菲举行。 近年来，在心脏发育、功能和心脏机制的理解方面取得了重大进展。 退行性疾病激发了人们对寻找新的治疗药物的兴趣，这些药物可能有助于治疗儿童和 患有心血管疾病的成人患者。然而，在细胞和动物模型中开发的治疗方法很少 可以很好地解释给人类患者。这次会议的目的是增进我们对心灵的理解 形成和稳态功能以及这些过程中的紊乱如何导致疾病和器官 功能障碍。由于人体心血管组织很难获得用于实验扰动，因此 会议将讨论一系列细胞和工程化组织模型的使用以及小型和大型 疾病的动物模型。本次会议还将展示最先进的工具和技术， 使心血管研究的各个领域发生了革命性的变化，包括心脏 发育生物学，心肌病和先天性心脏病遗传学，心脏组织工程， 以及细胞和再生疗法。这次活动将汇聚具有不同研究背景的科学家 世卫组织使用不同的工具和技术来研究心肌细胞和血管的机制 并将发现心血管生物学的新方法。该计划旨在 展示创新的、分子靶向的治疗罕见和常见心血管疾病的策略。

ABSTRACT Support is requested for a Keystone Symposia conference entitled Heart Development and Disease: From Genes to Cures, organized by Drs. Sean Wu, Wolfram Zimmermann and Christine Seidman. The conference will be held in Santa Fe, New Mexico from February 12-15, 2023. Significant advances in recent years in mechanistic understanding of cardiac development, function, and degeneration have stimulated interest in identifying novel therapeutic agents that may help treat children and adult patients with cardiovascular diseases. However, therapies developed in cell and animal models rarely translate well to human patients. The goals of this conference are to advance our understanding of heart formation and homeostatic function and how derangement in these processes lead to diseases and organ dysfunction. Since human cardiovascular tissue is difficult to obtain for experimental perturbation this conference will address the use of a spectrum of cells and engineered tissue models as well as small and large animal models of disease. This conference will also feature the state-of-the art tools and technologies that have revolutionized the developments in various domains of cardiovascular research including heart developmental biology, genetics of cardiomyopathy and congenital heart disease, cardiac tissue engineering, and cell and regenerative therapy. This event will bring together scientists with diverse research backgrounds who employ different tools and technologies to address mechanisms of cardiomyocyte and vascular dysfunction and will uncover new approaches to cardiovascular biology. The program is designed to showcase innovative, molecularly targeted strategies to treat both rare and common cardiovascular diseases.