The cancer-associated SF3B1 K700E spliceosome mutation confers enhanced sensitivity to BV-6-induced cytotoxicity

Abstract Recurrent somatic mutations in the key spliceosome component, SF3B1, have been identified at various frequencies across several cancer types. The most common hotspot mutation is the K700E missense mutation, and while its effects on splicing have been well characterised at the molecular leve...

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Main Authors: Lydia E. Roets, Jaine K. Blayney, Hayley P. McMillan, Patrick J. Preston, Alexander M. Mutch, Ken I. Mills, Kienan I. Savage, Katrina M. Lappin
Format: Article
Language:English
Published: Nature Publishing Group 2025-07-01
Series:Cell Death and Disease
Online Access:https://doi.org/10.1038/s41419-025-07790-y
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author Lydia E. Roets
Jaine K. Blayney
Hayley P. McMillan
Patrick J. Preston
Alexander M. Mutch
Ken I. Mills
Kienan I. Savage
Katrina M. Lappin
author_facet Lydia E. Roets
Jaine K. Blayney
Hayley P. McMillan
Patrick J. Preston
Alexander M. Mutch
Ken I. Mills
Kienan I. Savage
Katrina M. Lappin
author_sort Lydia E. Roets
collection DOAJ
description Abstract Recurrent somatic mutations in the key spliceosome component, SF3B1, have been identified at various frequencies across several cancer types. The most common hotspot mutation is the K700E missense mutation, and while its effects on splicing have been well characterised at the molecular level, the mis-spliced genes that contribute to cancer progression and/or dictate responses to therapy are still unclear. Here, we used we use cell line modelling to assess the impact of the SF3B1K700E mutation on the cellular response to various apoptosis-inducing agents. Our data suggest that the SF3B1K700E mutation leads to reduced cFLIP levels, along with defects in the splicing and translation of BCL2, causing a shift in the balance of pro- and anti-apoptotic genes and proteins, which confers greater sensitivity to the bivalent SMAC mimetic, BV-6. As such, BV-6 may represent a therapeutic opportunity for patients with SF3B1 mutant cancers.
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publisher Nature Publishing Group
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series Cell Death and Disease
spelling doaj-art-7939b4577d4c49b3872d2de833859e042025-08-20T04:01:36ZengNature Publishing GroupCell Death and Disease2041-48892025-07-0116111110.1038/s41419-025-07790-yThe cancer-associated SF3B1 K700E spliceosome mutation confers enhanced sensitivity to BV-6-induced cytotoxicityLydia E. Roets0Jaine K. Blayney1Hayley P. McMillan2Patrick J. Preston3Alexander M. Mutch4Ken I. Mills5Kienan I. Savage6Katrina M. Lappin7Patrick G Johnston Centre for Cancer Research, Queen’s University BelfastPatrick G Johnston Centre for Cancer Research, Queen’s University BelfastPatrick G Johnston Centre for Cancer Research, Queen’s University BelfastPatrick G Johnston Centre for Cancer Research, Queen’s University BelfastPatrick G Johnston Centre for Cancer Research, Queen’s University BelfastPatrick G Johnston Centre for Cancer Research, Queen’s University BelfastPatrick G Johnston Centre for Cancer Research, Queen’s University BelfastPatrick G Johnston Centre for Cancer Research, Queen’s University BelfastAbstract Recurrent somatic mutations in the key spliceosome component, SF3B1, have been identified at various frequencies across several cancer types. The most common hotspot mutation is the K700E missense mutation, and while its effects on splicing have been well characterised at the molecular level, the mis-spliced genes that contribute to cancer progression and/or dictate responses to therapy are still unclear. Here, we used we use cell line modelling to assess the impact of the SF3B1K700E mutation on the cellular response to various apoptosis-inducing agents. Our data suggest that the SF3B1K700E mutation leads to reduced cFLIP levels, along with defects in the splicing and translation of BCL2, causing a shift in the balance of pro- and anti-apoptotic genes and proteins, which confers greater sensitivity to the bivalent SMAC mimetic, BV-6. As such, BV-6 may represent a therapeutic opportunity for patients with SF3B1 mutant cancers.https://doi.org/10.1038/s41419-025-07790-y
spellingShingle Lydia E. Roets
Jaine K. Blayney
Hayley P. McMillan
Patrick J. Preston
Alexander M. Mutch
Ken I. Mills
Kienan I. Savage
Katrina M. Lappin
The cancer-associated SF3B1 K700E spliceosome mutation confers enhanced sensitivity to BV-6-induced cytotoxicity
Cell Death and Disease
title The cancer-associated SF3B1 K700E spliceosome mutation confers enhanced sensitivity to BV-6-induced cytotoxicity
title_full The cancer-associated SF3B1 K700E spliceosome mutation confers enhanced sensitivity to BV-6-induced cytotoxicity
title_fullStr The cancer-associated SF3B1 K700E spliceosome mutation confers enhanced sensitivity to BV-6-induced cytotoxicity
title_full_unstemmed The cancer-associated SF3B1 K700E spliceosome mutation confers enhanced sensitivity to BV-6-induced cytotoxicity
title_short The cancer-associated SF3B1 K700E spliceosome mutation confers enhanced sensitivity to BV-6-induced cytotoxicity
title_sort cancer associated sf3b1 k700e spliceosome mutation confers enhanced sensitivity to bv 6 induced cytotoxicity
url https://doi.org/10.1038/s41419-025-07790-y
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