The revolution in genetic engineering: CRISPR/Cas system Vol. 5, Núm. 2 Mayo-Agosto 2016 pp 116-128
Keywords:
CRISPR/Cas, gene therapy,, molecular biology, genetic expression, degenerative diseaseAbstract
CRISPRs (clustered regularly interspaced short palindromic repeat), along with the Cas endonuclease, form the CRISPR/Cas system. The system was discovered as a defense mechanism in bacteria and archaea, in which DNA from a pathogen —such as a bacteriophage— is incorporated between repeated palindromic sequences and later transcribed into an RNA known as crRNA. In a second infection, the crRNA coupled with Cas matches the pathogen’s transcript sequence and Cas silences or degrades the mRNA in a similar mechanism as a silencing RNA (siRNA). Due to its endonuclease activity and its ability to recognize specific sequences, the CRISPR/Cas system has been used in genetic engineering to activate or repress genes, to induce point mutations, and to alter sequences through homologous recombination. CRISPR has also been used to establish accurate models of human disease in mice and to evaluate cellular physiology through the simultaneous activation or repression of various genes. In this review article, we include the mechanism of action of the CRISPR/Cas system, its potential applications in cell and gene therapy, and future perspectives.
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Copyright (c) 2016 Instituto Nacional de Rehabilitación Luis Guillermo Ibarra Ibarra
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