Functional genomics and development of clinical genome editing strategies

戴维斯实验室的目标是了解血液和骨髓细胞如何读取基因组，以及如何编辑序列来治疗人类疾病。这一点很重要，因为血细胞中的遗传变异改变了许多不同人类疾病的易感性，包括感染、自身免疫性疾病和恶性肿瘤。这些细胞的基因组编辑有可能治愈许多疾病，但只有当我们了解改变基因组序列的影响时，这项技术才能安全地使用。超过90%的与人类疾病相关的DNA序列改变了基因的控制方式，而不是基因产生的蛋白质。该实验室在开发确定细胞内DNA物理结构的新技术方面拥有世界级的专业知识，这是了解基因组功能的关键。我们将使用这项技术来研究控制基因的基本原理。我们还将使用它来绘制控制重要血液和骨髓细胞类型基因的关键DNA序列。我们的目标是通过开发编辑骨髓干细胞基因组的新的、更安全的方法来翻译这一点，这些骨髓干细胞可以移植治疗人类疾病。

The Davies laboratory aims to understand how the genome is read by the cells of the blood and bone marrow and how the sequence can be edited to treat human disease. This is important because genetic variation in bloodcells alters the susceptibility to many different human diseases including infection, autoimmune disease and malignancy. Genome editing of these cells has the potential to cure many diseases but this technology can only be safely used if we understand the effects of altering the genome sequence. Over 90% of all DNA sequences that are associated with human disease alter how genes are controlled rather than in the protein produced by the gene. The laboratory has world class expertise in developing new techniques for determining the physical structure of DNA within cells, which is key to understanding how the genome functions. We will use this technology to investigate the fundamental principles that control genes. We will also use it to map the key DNA sequences that control genes in important blood and bone marrow cell types. We aim to translate this by developing new and safer ways of editing the genome of bone marrow stem cells which can be transplanted to treat human disease.