A goldilocks computational protocol for inhibitor discovery targeting DNA damage responses including replication-repair functions

While many researchers can design knockdown and knockout methodologies to remove a gene product, this is mainly untrue for new chemical inhibitor designs that empower multifunctional DNA Damage Response (DDR) networks. Here, we present a robust Goldilocks (GL) computational discovery protocol to eff...

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Main Authors: Davide Moiani, John A. Tainer
Format: Article
Language:English
Published: Frontiers Media S.A. 2024-11-01
Series:Frontiers in Molecular Biosciences
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Online Access:https://www.frontiersin.org/articles/10.3389/fmolb.2024.1442267/full
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author Davide Moiani
Davide Moiani
Davide Moiani
John A. Tainer
John A. Tainer
John A. Tainer
author_facet Davide Moiani
Davide Moiani
Davide Moiani
John A. Tainer
John A. Tainer
John A. Tainer
author_sort Davide Moiani
collection DOAJ
description While many researchers can design knockdown and knockout methodologies to remove a gene product, this is mainly untrue for new chemical inhibitor designs that empower multifunctional DNA Damage Response (DDR) networks. Here, we present a robust Goldilocks (GL) computational discovery protocol to efficiently innovate inhibitor tools and preclinical drug candidates for cellular and structural biologists without requiring extensive virtual screen (VS) and chemical synthesis expertise. By computationally targeting DDR replication and repair proteins, we exemplify the identification of DDR target sites and compounds to probe cancer biology. Our GL pipeline integrates experimental and predicted structures to efficiently discover leads, allowing early-structure and early-testing (ESET) experiments by many laboratories. By employing an efficient VS protocol to examine protein-protein interfaces (PPIs) and allosteric interactions, we identify ligand binding sites beyond active sites, leveraging in silico advances for molecular docking and modeling to screen PPIs and multiple targets. A diverse 3,174 compound ESET library combines Diamond Light Source DSI-poised, Protein Data Bank fragments, and FDA-approved drugs to span relevant chemotypes and facilitate downstream hit evaluation efficiency for academic laboratories. Two VS per library and multiple ranked ligand binding poses enable target testing for several DDR targets. This GL library and protocol can thus strategically probe multiple DDR network targets and identify readily available compounds for early structural and activity testing to overcome bottlenecks that can limit timely breakthrough drug discoveries. By testing accessible compounds to dissect multi-functional DDRs and suggesting inhibitor mechanisms from initial docking, the GL approach may enable more groups to help accelerate discovery, suggest new sites and compounds for challenging targets including emerging biothreats and advance cancer biology for future precision medicine clinical trials.
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spelling doaj-art-632138a147ec4d4d8690bbfa253a66fc2025-08-20T02:07:25ZengFrontiers Media S.A.Frontiers in Molecular Biosciences2296-889X2024-11-011110.3389/fmolb.2024.14422671442267A goldilocks computational protocol for inhibitor discovery targeting DNA damage responses including replication-repair functionsDavide Moiani0Davide Moiani1Davide Moiani2John A. Tainer3John A. Tainer4John A. Tainer5Department of Molecular and Cellular Oncology, University of Texas MD Anderson Cancer Center, Houston, TX, United StatesDepartment of Cancer Biology, University of Texas MD Anderson Cancer Center, Houston, TX, United StatesMoiani Research Inc., Missouri City, TX, United StatesDepartment of Molecular and Cellular Oncology, University of Texas MD Anderson Cancer Center, Houston, TX, United StatesDepartment of Cancer Biology, University of Texas MD Anderson Cancer Center, Houston, TX, United StatesBiophysics and Integrated Bioimaging, Lawrence Berkeley National Laboratory, Berkeley, CA, United StatesWhile many researchers can design knockdown and knockout methodologies to remove a gene product, this is mainly untrue for new chemical inhibitor designs that empower multifunctional DNA Damage Response (DDR) networks. Here, we present a robust Goldilocks (GL) computational discovery protocol to efficiently innovate inhibitor tools and preclinical drug candidates for cellular and structural biologists without requiring extensive virtual screen (VS) and chemical synthesis expertise. By computationally targeting DDR replication and repair proteins, we exemplify the identification of DDR target sites and compounds to probe cancer biology. Our GL pipeline integrates experimental and predicted structures to efficiently discover leads, allowing early-structure and early-testing (ESET) experiments by many laboratories. By employing an efficient VS protocol to examine protein-protein interfaces (PPIs) and allosteric interactions, we identify ligand binding sites beyond active sites, leveraging in silico advances for molecular docking and modeling to screen PPIs and multiple targets. A diverse 3,174 compound ESET library combines Diamond Light Source DSI-poised, Protein Data Bank fragments, and FDA-approved drugs to span relevant chemotypes and facilitate downstream hit evaluation efficiency for academic laboratories. Two VS per library and multiple ranked ligand binding poses enable target testing for several DDR targets. This GL library and protocol can thus strategically probe multiple DDR network targets and identify readily available compounds for early structural and activity testing to overcome bottlenecks that can limit timely breakthrough drug discoveries. By testing accessible compounds to dissect multi-functional DDRs and suggesting inhibitor mechanisms from initial docking, the GL approach may enable more groups to help accelerate discovery, suggest new sites and compounds for challenging targets including emerging biothreats and advance cancer biology for future precision medicine clinical trials.https://www.frontiersin.org/articles/10.3389/fmolb.2024.1442267/fullcancerDNA repairDNA damage responseDNA replicationprecision oncologycomputational pipeline
spellingShingle Davide Moiani
Davide Moiani
Davide Moiani
John A. Tainer
John A. Tainer
John A. Tainer
A goldilocks computational protocol for inhibitor discovery targeting DNA damage responses including replication-repair functions
Frontiers in Molecular Biosciences
cancer
DNA repair
DNA damage response
DNA replication
precision oncology
computational pipeline
title A goldilocks computational protocol for inhibitor discovery targeting DNA damage responses including replication-repair functions
title_full A goldilocks computational protocol for inhibitor discovery targeting DNA damage responses including replication-repair functions
title_fullStr A goldilocks computational protocol for inhibitor discovery targeting DNA damage responses including replication-repair functions
title_full_unstemmed A goldilocks computational protocol for inhibitor discovery targeting DNA damage responses including replication-repair functions
title_short A goldilocks computational protocol for inhibitor discovery targeting DNA damage responses including replication-repair functions
title_sort goldilocks computational protocol for inhibitor discovery targeting dna damage responses including replication repair functions
topic cancer
DNA repair
DNA damage response
DNA replication
precision oncology
computational pipeline
url https://www.frontiersin.org/articles/10.3389/fmolb.2024.1442267/full
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