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. 2021 Feb 4;10(2):118. doi: 10.3390/biology10020118

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© 2021 by the authors.

Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (http://creativecommons.org/licenses/by/4.0/).

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Conventional therapies vs. gene therapy for chronic myeloid leukemia (CML). Tyrosine-kinase inhibitor (TKI)-based conventional therapies are effective at silencing BCR/ABL1 in leukemic stem cells (LSCs). Treatment cessation can lead to relapse because of the existence of residual BCR/ABL1-positive cells. The appearance of TKI-resistant LSCs during treatment can lead to a relapse of the disease. However, anti-BCR/ABL1 gene therapy would eliminate the oncogene at the genome level. Corrected LSCs would be able to repopulate the bone marrow niche and thereby enable normal hematopoiesis.