Abstract
PARP inhibitors (PARPis) have been used to induce synthetic lethality in BRCA-deficient tumors in clinical trials with limited success. We hypothesized that RAD52-mediated DNA repair remains active in PARPi-treated BRCA-deficient tumor cells and that targeting RAD52 should enhance the synthetic lethal effect of PARPi. We show that RAD52 inhibitors (RAD52is) attenuated single-strand annealing (SSA) and residual homologous recombination (HR) in BRCA-deficient cells. Simultaneous targeting of PARP1 and RAD52 with inhibitors or dominant-negative mutants caused synergistic accumulation of DSBs and eradication of BRCA-deficient but not BRCA-proficient tumor cells. Remarkably, Parp1-/-;Rad52-/- mice are normal and display prolonged latency of BRCA1-deficient leukemia compared with Parp1-/- and Rad52-/- counterparts. Finally, PARPi+RAD52i exerted synergistic activity against BRCA1-deficient tumors in immunodeficient mice with minimal toxicity to normal cells and tissues. In conclusion, our data indicate that addition of RAD52i will improve therapeutic outcome of BRCA-deficient malignancies treated with PARPi.
Keywords:
BRCA-deficient tumors; PARP1; RAD52; synthetic lethality.
Copyright © 2018 The Author(s). Published by Elsevier Inc. All rights reserved.
Publication types
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Research Support, N.I.H., Extramural
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Research Support, Non-U.S. Gov't
MeSH terms
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Animals
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BRCA1 Protein / deficiency
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BRCA1 Protein / genetics*
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BRCA2 Protein / deficiency
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BRCA2 Protein / genetics*
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DNA Repair / drug effects
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Female
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Fusion Proteins, bcr-abl / genetics
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Fusion Proteins, bcr-abl / metabolism
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Homologous Recombination / drug effects
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Humans
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Imatinib Mesylate / pharmacology
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Kaplan-Meier Estimate
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Leukemia, Myeloid, Acute / metabolism
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Leukemia, Myeloid, Acute / mortality
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Leukemia, Myeloid, Acute / pathology
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Male
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Mice
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Mice, Inbred NOD
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Mice, Knockout
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Phthalazines / pharmacology
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Piperazines / pharmacology
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Poly (ADP-Ribose) Polymerase-1 / antagonists & inhibitors
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Poly (ADP-Ribose) Polymerase-1 / deficiency
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Poly (ADP-Ribose) Polymerase-1 / genetics*
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Rad52 DNA Repair and Recombination Protein / antagonists & inhibitors
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Rad52 DNA Repair and Recombination Protein / deficiency
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Rad52 DNA Repair and Recombination Protein / genetics*
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Synthetic Lethal Mutations
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Tumor Suppressor p53-Binding Protein 1 / deficiency
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Tumor Suppressor p53-Binding Protein 1 / genetics
Substances
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BRCA1 Protein
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BRCA1 protein, human
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BRCA2 Protein
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BRCA2 protein, human
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Phthalazines
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Piperazines
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Rad52 DNA Repair and Recombination Protein
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Rad52 protein, mouse
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Tumor Suppressor p53-Binding Protein 1
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Imatinib Mesylate
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Parp1 protein, mouse
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Poly (ADP-Ribose) Polymerase-1
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Fusion Proteins, bcr-abl
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olaparib