Targeting LLPS in disease: a new modality in drug development
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© 2023 Institute of Molecular Genetics and Genetic Engineering, University of Belgrade
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Biomolecular condensation is a process whereby many macromolecules (proteins and
RNAs) form non-stoichiometric, functional assemblies. The dominant mechanism of such
biomolecular condensation is liquid-liquid phase separation (LLPS), which leads to the
formation of membraneless organelles (MLOs), such as the nucleolus and stress granules,
in the cell. The proteins involved often have a high proportion of intrinsic structural
disorder, which drive LLPS by transient, multivalent interactions. As MLOs play key roles in
cell signaling, the misregulation of their formation and dissolution often leads to diseases
termed “condensatopathies”. In my presentation, I will outline the basic mechanisms
leading to such disease states, focusing on cancer, viral infections and neurodegeneration.
I will also discuss the different potential strategies for correcting these errors in cell
signaling, and show through specific examples how drug candidates, “c-mods” capable of
correcting MLO misre...gulation, can be developed.
Keywords:
LLPS databases / LLPS mechanism / LLPS targeting / condensatopathy / ALS/FTDSource:
4th Belgrade Bioinformatics Conference, 2023, 4, 4-4Publisher:
- Belgrade : Institute of molecular genetics and genetic engineering
Note:
- Book of abstract: 4th Belgrade Bioinformatics Conference, June 19-23, 2023
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Institut za molekularnu genetiku i genetičko inženjerstvoTY - CONF AU - Tompa, Peter PY - 2023 UR - https://belbi.bg.ac.rs/ UR - https://imagine.imgge.bg.ac.rs/handle/123456789/1939 AB - Biomolecular condensation is a process whereby many macromolecules (proteins and RNAs) form non-stoichiometric, functional assemblies. The dominant mechanism of such biomolecular condensation is liquid-liquid phase separation (LLPS), which leads to the formation of membraneless organelles (MLOs), such as the nucleolus and stress granules, in the cell. The proteins involved often have a high proportion of intrinsic structural disorder, which drive LLPS by transient, multivalent interactions. As MLOs play key roles in cell signaling, the misregulation of their formation and dissolution often leads to diseases termed “condensatopathies”. In my presentation, I will outline the basic mechanisms leading to such disease states, focusing on cancer, viral infections and neurodegeneration. I will also discuss the different potential strategies for correcting these errors in cell signaling, and show through specific examples how drug candidates, “c-mods” capable of correcting MLO misregulation, can be developed. PB - Belgrade : Institute of molecular genetics and genetic engineering C3 - 4th Belgrade Bioinformatics Conference T1 - Targeting LLPS in disease: a new modality in drug development EP - 4 SP - 4 VL - 4 UR - https://hdl.handle.net/21.15107/rcub_imagine_1939 ER -
@conference{ author = "Tompa, Peter", year = "2023", abstract = "Biomolecular condensation is a process whereby many macromolecules (proteins and RNAs) form non-stoichiometric, functional assemblies. The dominant mechanism of such biomolecular condensation is liquid-liquid phase separation (LLPS), which leads to the formation of membraneless organelles (MLOs), such as the nucleolus and stress granules, in the cell. The proteins involved often have a high proportion of intrinsic structural disorder, which drive LLPS by transient, multivalent interactions. As MLOs play key roles in cell signaling, the misregulation of their formation and dissolution often leads to diseases termed “condensatopathies”. In my presentation, I will outline the basic mechanisms leading to such disease states, focusing on cancer, viral infections and neurodegeneration. I will also discuss the different potential strategies for correcting these errors in cell signaling, and show through specific examples how drug candidates, “c-mods” capable of correcting MLO misregulation, can be developed.", publisher = "Belgrade : Institute of molecular genetics and genetic engineering", journal = "4th Belgrade Bioinformatics Conference", title = "Targeting LLPS in disease: a new modality in drug development", pages = "4-4", volume = "4", url = "https://hdl.handle.net/21.15107/rcub_imagine_1939" }
Tompa, P.. (2023). Targeting LLPS in disease: a new modality in drug development. in 4th Belgrade Bioinformatics Conference Belgrade : Institute of molecular genetics and genetic engineering., 4, 4-4. https://hdl.handle.net/21.15107/rcub_imagine_1939
Tompa P. Targeting LLPS in disease: a new modality in drug development. in 4th Belgrade Bioinformatics Conference. 2023;4:4-4. https://hdl.handle.net/21.15107/rcub_imagine_1939 .
Tompa, Peter, "Targeting LLPS in disease: a new modality in drug development" in 4th Belgrade Bioinformatics Conference, 4 (2023):4-4, https://hdl.handle.net/21.15107/rcub_imagine_1939 .