Genome editing with CRISPR-Cas9 and its application in genetic disorders

Authors

  • Riber Fabián Donoso Noroña
  • Nairovys Gómez Martínez
  • Adisnay Rodríguez Plasencia

Keywords:

genome editing, CRISPR-Cas9, genetic disorders, gene therapy, genetic mutations.

Abstract

Genome editing with CRISPR-Cas9 has emerged as a revolutionary tool in biomedical research, allowing precise manipulation of genes in a wide variety of organisms. One of the most promising fields of application of this technology is the approach to genetic disorders. The main objective of this literature review is to provide a comprehensive overview of the current achievements and areas of development of genome editing in the context of genetic disorders. The aim is to evaluate the effectiveness of genomic editing strategies and explore the possible clinical implications of this technology in the therapy of genetic diseases. An exhaustive search of current scientific literature was carried out in academic databases. Preclinical and clinical studies related to the application of CRISPR-Cas9 in genetic disorders were identified. The methodological approaches used and the results obtained were analyzed. The results highlight significant growth in the efficiency and precision of genome editing, as well as advances in adapting this technology to specific genetic disorders. There is an increase in clinical trials and promising therapeutic approaches. Genome editing with CRISPR-Cas9 represents a powerful strategy to correct genetic mutations underlying inherited disorders. Although technical and ethical challenges exist, current advances suggest a promising future for precision gene therapy.

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Published

2024-06-16

How to Cite

1.
Donoso Noroña RF, Gómez Martínez N, Rodríguez Plasencia A. Genome editing with CRISPR-Cas9 and its application in genetic disorders. Rev Cubana Inv Bioméd [Internet]. 2024 Jun. 16 [cited 2025 Aug. 2];42(2). Available from: https://revibiomedica.sld.cu/index.php/ibi/article/view/3354