Fundamentos da terapia gênica: explorando as estratégias terapêuticas da revolução genética

• DOI: https://doi.org/10.22533/at.ed.3812516019

• Palavras-chave: Terapia gênica. Doenças genéticas. CRISPR-Cas9. Vetores. Edição gênica.

• Keywords: Gene therapy. Genetic diseases. CRISPR-Cas9. Vectors. Gene editing.

• Abstract: Gene therapy is an innovative approach for treating genetic diseases and other medical conditions, offering potentially curative solutions for diseases previously considered incurable or difficult to manage. Currently, there are four primary types of gene therapy: supplementation, silencing, replacement, and editing. Gene therapy can be conducted using various techniques, such as viral vectors, bacterial vectors, RNA interference, and CRISPR-Cas9. One of the main advantages of this approach is its ability to address the genetic root cause of a condition, eliminating the need for ongoing medications and therapies, unlike conventional treatments that often require repeated infusions. Despite significant advancements in this field, challenges remain, such as the risk of unintended effects (off-target effects) and limited access to this type of therapeutic approach for the majority of the world's population. Thus, while gene therapy opens up vast possibilities for treating conditions once considered permanent, further studies are necessary to better understand and mitigate off-target effects. Additionally, efforts to reduce production costs and improve accessibility in less developed countries are essential to ensure the broader availability of these groundbreaking treatments.