Modeling Transient Abnormal Myelopoiesis Using Induced Pluripotent Stem Cells and CRISPR/Cas9 Technology
Approximately 1%–2% of children with Down syndrome (DS) develop acute myeloid leukemia (AML) prior to age 5 years. AML in DS children (ML-DS) is characterized by the pathognomonic mutation in the gene encoding the essential hematopoietic transcription factor GATA1, resulting in N-terminally truncate...
Main Authors: | , , , |
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Format: | Article |
Language: | English |
Published: |
Elsevier
2020-12-01
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Series: | Molecular Therapy: Methods & Clinical Development |
Subjects: | |
Online Access: | http://www.sciencedirect.com/science/article/pii/S2329050120301923 |