Realities and perspectives of the use of gene therapy in the treatment of diseases
DOI:
https://doi.org/10.23925/1984-4840.2018v20i3a2Keywords:
gene transfer techniques, genetic therapy, genetic engineering, genetic services, CRISPR-Cas systemsAbstract
This update presents the most important events regarding the applicability of gene therapy in humans. Gene therapy is a therapeutic modality for single gene or polygenic genetic diseases and acquired diseases. Gene therapy is a clinical intervention method based on genetic engineering techniques, which intentionally modifies the genome of some cells. Gene manipulation occurs with the purpose of editing and/or inserting transgenes to supply an inactive or dysfunctional gene. In this update, we describe the emergence of the first ideas about this therapy, we demonstrate the types of biotechnological interventions and some methods for genes transference through viral and non-viral vectors and the promising CRISPR/Cas9 system. We also report the main types of diseases which can be treated through this procedure and we analyze limitations and possible risks associated with the use of this therapy. We conclude by exposing the current realities of clinical trials in Brazil and in the world.Downloads
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