CRISPR/CAS9 gene editing in diabetes mellitus: a future cure


  • Cristóbal Espinoza
  • Alicia Morocho
  • Eliberth Morales
  • Marlon López
  • María Paula Calderón
  • Zoila del Cisne Espinoza

Palabras clave:

Diabetes mellitus, CRISPR-Cas9, gene therapy, gene editing, diabetes therapy


Current advances in gene therapy have shown great potential in experimental studies for several disorders, including diabetes mellitus (DM). Site-specific gene editing has been enabled by clustered regularly interspaced short palindromic repeats (CRISPR) and its CRISPR-associated protein 9 (Cas9), a third-generation nuclease that causes double-strand DNA to break. This process allows the insertion, deletion or silencing of any desired gene. Studies in mice have demonstrated that diabetic status can be reversed by specific gene targeting. Other studies have targeted obesity and obesity-related genes as an alternative to improve insulin resistance and provide better metabolic homeostasis. There is ongoing investigation to identify the best possible strategy to treat DM with the CRISPR system. This review aims to analyze different experimental gene therapy studies mediated by CRISPR-Cas9 to determine their efficacy, safety profile and reliability as a possible future cure for DM. Likewise, basic concepts regarding CRISPR/cas9 will be reviewed.


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