Preclinical studies in cell cultures and animals, as well as clinical trials in humans, are an essential part of the development of new drugs or therapies because they determine safety, feasibility and efficacy, while also indicating potential adverse side effects of the therapies. Since its designation as a gene-editing tool in 2012, CRISPR technology has made significant breakthroughs. It is a relatively simple editing technology in which the target site in the genome is determined by the sequence of the single guide RNA molecule (sgRNA). The technology has been rapidly applied to treat numerous human genetic diseases. Numerous preclinical studies are currently underway to test the treatment potential for various diseases, such as cancer, cystic fibrosis, muscular dystrophy and many others. Therapies that have been successful in the preclinical phase have made their way into clinical trials, including the treatment of sickle cell anemia and the treatment of blindness. Most of the clinical trials are still ongoing, meaning that the results have not yet been published. Based on the results of the clinical trials, treatment of sickle cell anemia and beta thalassemia seems most likely in the near future. Promising examples of CRISPR-based treatments include the case of Victoria Gray and the preliminary results in the treatment of blindness. In my bachelor thesis I aimed to show for which diseases CRISPR-based treatments are most developed and to summarize the main results of preclinical and clinical studies.
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