BMC Biotechnology (Jul 2018)

Targeted genome editing in acute lymphoblastic leukemia: a review

  • Adrián Montaño,
  • Maribel Forero-Castro,
  • Jesús-María Hernández-Rivas,
  • Ignacio García-Tuñón,
  • Rocío Benito

DOI
https://doi.org/10.1186/s12896-018-0455-9
Journal volume & issue
Vol. 18, no. 1
pp. 1 – 10

Abstract

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Abstract Background Genome editing technologies offers new opportunities for tackling diseases such as acute lymphoblastic leukemia (ALL) that have been beyond the reach of previous therapies. Results We show how the recent availability of genome-editing tools such as CRISPR-Cas9 are an important means of advancing functional studies of ALL through the incorporation, elimination and modification of somatic mutations and fusion genes in cell lines and mouse models. These tools not only broaden the understanding of the involvement of various genetic alterations in the pathogenesis of the disease but also identify new therapeutic targets for future clinical trials. Conclusions New approaches including CRISPR-Cas9 are crucial for functional studies of genetic aberrations driving cancer progression, and that may be responsible for treatment resistance and relapses. By using this approach, diseases can be more faithfully reproduced and new therapeutic targets and approaches found.

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