A biochemical screening platform to target chromatin states using condensates as a tool

Chromatin states define cell fates and consequently dysfunctional chromatin states drive disease. Conventional approaches to target dysfunctional chromatin states typically rely on targeting a defined, structured binding pocket of a specific chromatin protein. However, drugs developed from targeting...

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Main Authors: Laura J. Hsieh, Tracy Lou, Muryam A. Gourdet, Emily Wong, Geeta J. Narlikar
Format: Article
Language:English
Published: Elsevier 2025-06-01
Series:SLAS Discovery
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Online Access:http://www.sciencedirect.com/science/article/pii/S2472555225000292
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author Laura J. Hsieh
Tracy Lou
Muryam A. Gourdet
Emily Wong
Geeta J. Narlikar
author_facet Laura J. Hsieh
Tracy Lou
Muryam A. Gourdet
Emily Wong
Geeta J. Narlikar
author_sort Laura J. Hsieh
collection DOAJ
description Chromatin states define cell fates and consequently dysfunctional chromatin states drive disease. Conventional approaches to target dysfunctional chromatin states typically rely on targeting a defined, structured binding pocket of a specific chromatin protein. However, drugs developed from targeting single chromatin proteins have often failed in the clinic due to toxicity from broad non-specific effects on the genome. Substantial previous work has indicated that the function of a given chromatin state is encoded in the context-dependent protein-protein interactions (PPIs) between the Intrinsically disordered regions (IDRs) and folded domains of the multiple constituents. Currently, there are no drug discovery approaches that target the complex multivalent protein interactions within a given dysfunctional chromatin state. Therefore, new methods are required to target chromatin within specific conformational contexts for better translation into humans. Prior discoveries from our group and others have shown that chromatin intrinsically forms condensates through weak, yet specific, multivalent interactions between itself and other components. Using this intrinsic property of chromatin, we have developed a new screening method to address this technology gap and identify modulators of dysfunctional chromatin states for drug discovery. Here, we show that we can recreate different chromatin contexts as phase-separated condensates that have distinct biochemical and biophysical properties. Furthermore, we have scaled the technology into a screening platform and identify small molecules that modulate chromatin states specifically based on their chromatin context. We anticipate that such specific targeting of a disease driving chromatin assembly would reduce off-target effects, translate better into humans and open a new landscape of therapeutic possibilities for targeting complex, multivalent interactions.
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spelling doaj-art-e1b7e9e024874824a3f1a03d0843e86b2025-08-20T03:49:03ZengElsevierSLAS Discovery2472-55522025-06-013310023610.1016/j.slasd.2025.100236A biochemical screening platform to target chromatin states using condensates as a toolLaura J. Hsieh0Tracy Lou1Muryam A. Gourdet2Emily Wong3Geeta J. Narlikar4Department of Biochemistry and Biophysics, University of California, San Francisco, San Francisco, CA 94158, USA; TippingPoint Biosciences, Inc., San Francisco, CA 94107, USA; Lead Contacts, USA; Corresponding author at: TippingPoint Biosciences, Inc., San Francisco, CA 94107, USA.Department of Biochemistry and Biophysics, University of California, San Francisco, San Francisco, CA 94158, USADepartment of Biochemistry and Biophysics, University of California, San Francisco, San Francisco, CA 94158, USA; Tetrad Graduate Program, University of California, San Francisco, San Francisco, CA 94158, USA; TippingPoint Biosciences, Inc., San Francisco, CA 94107, USADepartment of Biochemistry and Biophysics, University of California, San Francisco, San Francisco, CA 94158, USADepartment of Biochemistry and Biophysics, University of California, San Francisco, San Francisco, CA 94158, USA; Lead Contacts, USA; Corresponding author at: Department of Biochemistry and Biophysics, University of California, San Francisco, San Francisco, CA 94158, USA.Chromatin states define cell fates and consequently dysfunctional chromatin states drive disease. Conventional approaches to target dysfunctional chromatin states typically rely on targeting a defined, structured binding pocket of a specific chromatin protein. However, drugs developed from targeting single chromatin proteins have often failed in the clinic due to toxicity from broad non-specific effects on the genome. Substantial previous work has indicated that the function of a given chromatin state is encoded in the context-dependent protein-protein interactions (PPIs) between the Intrinsically disordered regions (IDRs) and folded domains of the multiple constituents. Currently, there are no drug discovery approaches that target the complex multivalent protein interactions within a given dysfunctional chromatin state. Therefore, new methods are required to target chromatin within specific conformational contexts for better translation into humans. Prior discoveries from our group and others have shown that chromatin intrinsically forms condensates through weak, yet specific, multivalent interactions between itself and other components. Using this intrinsic property of chromatin, we have developed a new screening method to address this technology gap and identify modulators of dysfunctional chromatin states for drug discovery. Here, we show that we can recreate different chromatin contexts as phase-separated condensates that have distinct biochemical and biophysical properties. Furthermore, we have scaled the technology into a screening platform and identify small molecules that modulate chromatin states specifically based on their chromatin context. We anticipate that such specific targeting of a disease driving chromatin assembly would reduce off-target effects, translate better into humans and open a new landscape of therapeutic possibilities for targeting complex, multivalent interactions.http://www.sciencedirect.com/science/article/pii/S2472555225000292ChromatinCondensatesPPIsIDRs
spellingShingle Laura J. Hsieh
Tracy Lou
Muryam A. Gourdet
Emily Wong
Geeta J. Narlikar
A biochemical screening platform to target chromatin states using condensates as a tool
SLAS Discovery
Chromatin
Condensates
PPIs
IDRs
title A biochemical screening platform to target chromatin states using condensates as a tool
title_full A biochemical screening platform to target chromatin states using condensates as a tool
title_fullStr A biochemical screening platform to target chromatin states using condensates as a tool
title_full_unstemmed A biochemical screening platform to target chromatin states using condensates as a tool
title_short A biochemical screening platform to target chromatin states using condensates as a tool
title_sort biochemical screening platform to target chromatin states using condensates as a tool
topic Chromatin
Condensates
PPIs
IDRs
url http://www.sciencedirect.com/science/article/pii/S2472555225000292
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