Enhancer Hijacking Drives Oncogenic BCL11B Expression in Lineage-Ambiguous Stem Cell Leukemia

Lineage-ambiguous leukemias are high-risk malignancies of poorly understood genetic basis. Here, we describe a distinct subgroup of acute leukemia with expression of myeloid, T lymphoid, and stem cell markers driven by aberrant allele-specific deregulation of , a master transcription factor responsi...

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Vydáno v:Cancer discovery Ročník 11; číslo 11; s. 2846
Hlavní autoři: Montefiori, Lindsey E, Bendig, Sonja, Gu, Zhaohui, Chen, Xiaolong, Pölönen, Petri, Ma, Xiaotu, Murison, Alex, Zeng, Andy, Garcia-Prat, Laura, Dickerson, Kirsten, Iacobucci, Ilaria, Abdelhamed, Sherif, Hiltenbrand, Ryan, Mead, Paul E, Mehr, Cyrus M, Xu, Beisi, Cheng, Zhongshan, Chang, Ti-Cheng, Westover, Tamara, Ma, Jing, Stengel, Anna, Kimura, Shunsuke, Qu, Chunxu, Valentine, Marcus B, Rashkovan, Marissa, Luger, Selina, Litzow, Mark R, Rowe, Jacob M, den Boer, Monique L, Wang, Victoria, Yin, Jun, Kornblau, Steven M, Hunger, Stephen P, Loh, Mignon L, Pui, Ching-Hon, Yang, Wenjian, Crews, Kristine R, Roberts, Kathryn G, Yang, Jun J, Relling, Mary V, Evans, William E, Stock, Wendy, Paietta, Elisabeth M, Ferrando, Adolfo A, Zhang, Jinghui, Kern, Wolfgang, Haferlach, Torsten, Wu, Gang, Dick, John E, Klco, Jeffery M, Haferlach, Claudia, Mullighan, Charles G
Médium: Journal Article
Jazyk:angličtina
Vydáno: United States 01.11.2021
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ISSN:2159-8290, 2159-8290
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Abstract Lineage-ambiguous leukemias are high-risk malignancies of poorly understood genetic basis. Here, we describe a distinct subgroup of acute leukemia with expression of myeloid, T lymphoid, and stem cell markers driven by aberrant allele-specific deregulation of , a master transcription factor responsible for thymic T-lineage commitment and specification. Mechanistically, this deregulation was driven by chromosomal rearrangements that juxtapose to superenhancers active in hematopoietic progenitors, or focal amplifications that generate a superenhancer from a noncoding element distal to . Chromatin conformation analyses demonstrated long-range interactions of rearranged enhancers with the expressed allele and association of with activated hematopoietic progenitor cell -regulatory elements, suggesting BCL11B is aberrantly co-opted into a gene regulatory network that drives transformation by maintaining a progenitor state. These data support a role for ectopic expression in primitive hematopoietic cells mediated by enhancer hijacking as an oncogenic driver of human lineage-ambiguous leukemia. SIGNIFICANCE: Lineage-ambiguous leukemias pose significant diagnostic and therapeutic challenges due to a poorly understood molecular and cellular basis. We identify oncogenic deregulation of driven by diverse structural alterations, including superenhancer generation, as the driving feature of a subset of lineage-ambiguous leukemias that transcend current diagnostic boundaries. .
AbstractList Lineage-ambiguous leukemias are high-risk malignancies of poorly understood genetic basis. Here, we describe a distinct subgroup of acute leukemia with expression of myeloid, T lymphoid, and stem cell markers driven by aberrant allele-specific deregulation of BCL11B, a master transcription factor responsible for thymic T-lineage commitment and specification. Mechanistically, this deregulation was driven by chromosomal rearrangements that juxtapose BCL11B to superenhancers active in hematopoietic progenitors, or focal amplifications that generate a superenhancer from a noncoding element distal to BCL11B. Chromatin conformation analyses demonstrated long-range interactions of rearranged enhancers with the expressed BCL11B allele and association of BCL11B with activated hematopoietic progenitor cell cis-regulatory elements, suggesting BCL11B is aberrantly co-opted into a gene regulatory network that drives transformation by maintaining a progenitor state. These data support a role for ectopic BCL11B expression in primitive hematopoietic cells mediated by enhancer hijacking as an oncogenic driver of human lineage-ambiguous leukemia. SIGNIFICANCE: Lineage-ambiguous leukemias pose significant diagnostic and therapeutic challenges due to a poorly understood molecular and cellular basis. We identify oncogenic deregulation of BCL11B driven by diverse structural alterations, including de novo superenhancer generation, as the driving feature of a subset of lineage-ambiguous leukemias that transcend current diagnostic boundaries.This article is highlighted in the In This Issue feature, p. 2659.Lineage-ambiguous leukemias are high-risk malignancies of poorly understood genetic basis. Here, we describe a distinct subgroup of acute leukemia with expression of myeloid, T lymphoid, and stem cell markers driven by aberrant allele-specific deregulation of BCL11B, a master transcription factor responsible for thymic T-lineage commitment and specification. Mechanistically, this deregulation was driven by chromosomal rearrangements that juxtapose BCL11B to superenhancers active in hematopoietic progenitors, or focal amplifications that generate a superenhancer from a noncoding element distal to BCL11B. Chromatin conformation analyses demonstrated long-range interactions of rearranged enhancers with the expressed BCL11B allele and association of BCL11B with activated hematopoietic progenitor cell cis-regulatory elements, suggesting BCL11B is aberrantly co-opted into a gene regulatory network that drives transformation by maintaining a progenitor state. These data support a role for ectopic BCL11B expression in primitive hematopoietic cells mediated by enhancer hijacking as an oncogenic driver of human lineage-ambiguous leukemia. SIGNIFICANCE: Lineage-ambiguous leukemias pose significant diagnostic and therapeutic challenges due to a poorly understood molecular and cellular basis. We identify oncogenic deregulation of BCL11B driven by diverse structural alterations, including de novo superenhancer generation, as the driving feature of a subset of lineage-ambiguous leukemias that transcend current diagnostic boundaries.This article is highlighted in the In This Issue feature, p. 2659.
Lineage-ambiguous leukemias are high-risk malignancies of poorly understood genetic basis. Here, we describe a distinct subgroup of acute leukemia with expression of myeloid, T lymphoid, and stem cell markers driven by aberrant allele-specific deregulation of , a master transcription factor responsible for thymic T-lineage commitment and specification. Mechanistically, this deregulation was driven by chromosomal rearrangements that juxtapose to superenhancers active in hematopoietic progenitors, or focal amplifications that generate a superenhancer from a noncoding element distal to . Chromatin conformation analyses demonstrated long-range interactions of rearranged enhancers with the expressed allele and association of with activated hematopoietic progenitor cell -regulatory elements, suggesting BCL11B is aberrantly co-opted into a gene regulatory network that drives transformation by maintaining a progenitor state. These data support a role for ectopic expression in primitive hematopoietic cells mediated by enhancer hijacking as an oncogenic driver of human lineage-ambiguous leukemia. SIGNIFICANCE: Lineage-ambiguous leukemias pose significant diagnostic and therapeutic challenges due to a poorly understood molecular and cellular basis. We identify oncogenic deregulation of driven by diverse structural alterations, including superenhancer generation, as the driving feature of a subset of lineage-ambiguous leukemias that transcend current diagnostic boundaries. .
Author Westover, Tamara
Kern, Wolfgang
Relling, Mary V
Luger, Selina
Crews, Kristine R
Yang, Jun J
Yin, Jun
Haferlach, Torsten
Kornblau, Steven M
Mead, Paul E
Dickerson, Kirsten
Wu, Gang
Chen, Xiaolong
den Boer, Monique L
Wang, Victoria
Paietta, Elisabeth M
Hunger, Stephen P
Pölönen, Petri
Roberts, Kathryn G
Chang, Ti-Cheng
Rashkovan, Marissa
Rowe, Jacob M
Ma, Xiaotu
Cheng, Zhongshan
Valentine, Marcus B
Mehr, Cyrus M
Haferlach, Claudia
Qu, Chunxu
Zhang, Jinghui
Garcia-Prat, Laura
Yang, Wenjian
Iacobucci, Ilaria
Dick, John E
Ferrando, Adolfo A
Montefiori, Lindsey E
Stengel, Anna
Kimura, Shunsuke
Gu, Zhaohui
Xu, Beisi
Stock, Wendy
Klco, Jeffery M
Pui, Ching-Hon
Evans, William E
Bendig, Sonja
Abdelhamed, Sherif
Murison, Alex
Hiltenbrand, Ryan
Ma, Jing
Zeng, Andy
Loh, Mignon L
Litzow, Mark R
Mullighan, Charles G
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  organization: Munich Leukemia Laboratory, Munich, Germany
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  organization: Department of Molecular Genetics, University of Toronto, Toronto, Canada
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  organization: Department of Molecular Genetics, University of Toronto, Toronto, Canada
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  organization: Department of Pathology, St. Jude Children's Research Hospital, Memphis, Tennessee
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  organization: Department of Pathology, St. Jude Children's Research Hospital, Memphis, Tennessee. charles.mullighan@stjude.org Jeffery.klco@stjude.org Claudia.haferlach@mll.com
BackLink https://www.ncbi.nlm.nih.gov/pubmed/34103329$$D View this record in MEDLINE/PubMed
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PublicationTitle Cancer discovery
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Snippet Lineage-ambiguous leukemias are high-risk malignancies of poorly understood genetic basis. Here, we describe a distinct subgroup of acute leukemia with...
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SubjectTerms Enhancer Elements, Genetic
Gene Regulatory Networks
Hematopoietic Stem Cells - metabolism
Humans
Leukemia, Myeloid, Acute - genetics
Leukemia, Myeloid, Acute - metabolism
Repressor Proteins - biosynthesis
Repressor Proteins - genetics
Repressor Proteins - metabolism
Transcription Factors - genetics
Tumor Suppressor Proteins - biosynthesis
Tumor Suppressor Proteins - genetics
Title Enhancer Hijacking Drives Oncogenic BCL11B Expression in Lineage-Ambiguous Stem Cell Leukemia
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