Gene editing is a genetic engineering technique in which a DNA sequence is modified, deleted or inserted into the genome of any type of living organism. Using this technique, it is possible to study the causes of different diseases and find new therapeutic alternatives.
At LIAN-FLENI-CONICET we are working on gene editing of stem cells with different objectives including the study of specific proteins to understand their function during cardiac development, as well as the modeling of genetic diseases for the development of new therapies. For disease modeling, we work with samples from patients with a genetic disease, but we also generate small point mutations or large deletions or insertions in healthy genomes in the laboratory to reproduce a disease and study its phenotype in vitro or in vivo. In the first case, it is possible to develop a targeted therapy for the patient and revert the disease to a healthy genotype with gene-editing techniques. In the second, it is possible to induce a genetic disease, characterize it in vitro and then develop possible drugs evaluating their toxicological and therapeutic effect without putting the health of patients at risk. The projects we develop at LIAN-FLENI-CONICET have great implications in their areas of application, including the development of new drugs and precision medicine. The possibility of modeling and characterize a genetic disease in vitro is of great relevance in order to generate knowledge and advanced technologies in health.
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