Development of novel therapies for genetic diseases using the Gentrafix platform

Bitrobius Genetics的目标是开发三种重要和普遍的遗传病的治疗方法：囊性纤维化、肌营养不良和先天性肌强直，以及遗传性乳腺癌。我们将使用一种名为Gentrafix的新技术来实现这一点，该技术将极大地提高DNA使用基因编辑技术修复有缺陷基因的能力。它可以携带比目前技术所能携带的更大的基因，并且只需一次应用，就能够到达受影响器官的大多数细胞。第一次，目前没有治疗方法的遗传病将能够被治愈，导致或正在积极导致癌症的突变可以得到纠正。生产成本将低于任何竞争技术。基因由DNA组成，代表制造蛋白质的指令，蛋白质反过来形成结构并在体内执行功能。遗传性疾病和癌症是由含有错误的基因引起的，在遗传性疾病的情况下会导致残疾，在癌症的情况下会导致细胞生长失控。基因治疗是一种旨在纠正这些遗传错误的技术。不幸的是，基因疗法未能治愈大多数遗传性疾病，现存的为数不多的疗法是历史上最昂贵的药物。根本的问题是，没有可靠的方法将正确的DNA送到必须修复的器官中的每个细胞。大多数基因疗法使用的是经过改造的病毒，这种病毒只能感染少数目标细胞，不能携带大基因，在细胞之间传播的能力有限，可以被免疫系统灭活，必须在患者的一生中持续给药。项目团队将构建疾病治疗方案，并在实验室实验中对其进行测试，这样在项目结束时，四名主要候选者将准备开始在人体上进行测试的过程。

Bitrobius Genetics aims to develop cures for three important and prevalent genetic diseases: cystic fibrosis, muscular dystrophy and myotonia congenita, as well as hereditary breast cancer. We will do this using a new technology called Gentrafix, which will greatly improve the ability of DNA to repair faulty genes using a gene editing technology. It can transport much larger genes than can be carried using current technologies and will be able to reach most of the cells in an affected organ with a single application. For the first time, genetic diseases with no current treatment will be able to be cured, and mutations that lead to or are actively causing cancers can be corrected. The production costs will be lower than those of any competing technology.Genes are made of DNA and represent instructions for making proteins, which in turn form the structure and carry out functions within the body. Genetic diseases and cancers are caused by genes that contain errors, leading to disability in the case of genetic diseases or uncontrolled cell growth in the case of cancers. Gene therapy is a technology that aims to correct these genetic errors. Unfortunately, gene therapy has failed to cure most genetic diseases, and the few therapies that do exist are the most expensive medicines in history. The fundamental problem is that there is no reliable way of getting the correct DNA to every cell in an organ that must be fixed. Most gene therapies use modified viruses which can only infect a few percent of target cells, cannot carry large genes, have a limited ability to spread between cells, can be inactivated by the immune system and must be administered continuously during a patient's life.The project team will construct the disease cures and test them in laboratory experiments, such that at the end of the project, the four primary candidates will be ready to begin the process leading to testing in humans.