Preprint Review Version 1 Preserved in Portico This version is not peer-reviewed

Future Approaches for Treating Chronic Myeloid Leukemia: CRISPR Therapy

Version 1 : Received: 11 January 2021 / Approved: 12 January 2021 / Online: 12 January 2021 (10:14:46 CET)

A peer-reviewed article of this Preprint also exists.

Vuelta, E.; García-Tuñón, I.; Hernández-Carabias, P.; Méndez, L.; Sánchez-Martín, M. Future Approaches for Treating Chronic Myeloid Leukemia: CRISPR Therapy. Biology 2021, 10, 118. Vuelta, E.; García-Tuñón, I.; Hernández-Carabias, P.; Méndez, L.; Sánchez-Martín, M. Future Approaches for Treating Chronic Myeloid Leukemia: CRISPR Therapy. Biology 2021, 10, 118.

Journal reference: Biology 2021, 10, 118
DOI: 10.3390/biology10020118

Abstract

The constitutively active tyrosine kinase BCR/ABL1 oncogene plays a key role in human chronic myeloid leukemia development and disease maintenance, and determines most of the features of this leukemia. For this reason, tyrosine kinase inhibitors are the first-line treatment, offering most patients a life expectancy like that of an equivalent healthy person. However, since the oncogene is not destroyed, lifelong oral medication is essential, even though this trigger adverse effects in many patients. Furthermore, leukemic stem cells remain quiescent and resistance is observed in approximately 25% of patients. Thus, new therapeutic alternatives are still needed. In this scenario, the emergence of CRISPR technology can offer a definitive treatment based on its capacity to disrupt coding sequences. This review describes CML disease and the main advances in the genome-editing field by which it may be treated in the future.

Keywords

CML; CRISPR/Cas9; BCR/ABL1; genome editing

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