GENE THERAPIES AND CRISPR IN CLINICAL MEDICINE: APPLICATIONS, ADVANCES AND ETHICAL CHALLENGES

• DOI: https://doi.org/10.22533/at.ed.15941012402126

• Palavras-chave: CRISPR-Cas9, Gene Therapy, Personalized Medicine, Muscular Dystrophy, Sickle Cell Anemia, Ethics and Regulation.

• Keywords: CRISPR-Cas9, Gene Therapy, Personalized Medicine, Muscular Dystrophy, Sickle Cell Anemia, Ethics and Regulation.

• Abstract:

  Gene editing using the CRISPR-Cas9 technique has emerged as one of the greatest innovations in clinical medicine, offering the potential to correct genetic mutations responsible for various diseases. This technology has been applied in the treatment of rare and common diseases such as muscular dystrophy, sickle cell anemia and cancer, bringing new paradigms to personalized medicine. This article addresses the clinical applications of CRISPR-Cas9, exploring its possibilities, challenges and ethical and regulatory implications. In addition, it discusses the advancement of personalized therapies, with a focus on clinical feasibility, accessibility and the obstacles faced for the widespread adoption of these therapies. The study also highlights the regulatory aspects that need to be adjusted to ensure the safety and efficacy of these innovative approaches.