De novo-designed transmembrane proteins bind and regulate a cytokine receptor.


Journal

Nature chemical biology
ISSN: 1552-4469
Titre abrégé: Nat Chem Biol
Pays: United States
ID NLM: 101231976

Informations de publication

Date de publication:
13 Mar 2024
Historique:
received: 14 02 2023
accepted: 25 01 2024
medline: 14 3 2024
pubmed: 14 3 2024
entrez: 14 3 2024
Statut: aheadofprint

Résumé

Transmembrane (TM) domains as simple as a single span can perform complex biological functions using entirely lipid-embedded chemical features. Computational design has the potential to generate custom tool molecules directly targeting membrane proteins at their functional TM regions. Thus far, designed TM domain-targeting agents have been limited to mimicking the binding modes and motifs of natural TM interaction partners. Here, we demonstrate the design of de novo TM proteins targeting the erythropoietin receptor (EpoR) TM domain in a custom binding topology competitive with receptor homodimerization. The TM proteins expressed in mammalian cells complex with EpoR and inhibit erythropoietin-induced cell proliferation. In vitro, the synthetic TM domain complex outcompetes EpoR homodimerization. Structural characterization reveals that the complex involves the intended amino acids and agrees with our designed molecular model of antiparallel TM helices at 1:1 stoichiometry. Thus, membrane protein TM regions can now be targeted in custom-designed topologies.

Identifiants

pubmed: 38480980
doi: 10.1038/s41589-024-01562-z
pii: 10.1038/s41589-024-01562-z
doi:

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Subventions

Organisme : U.S. Department of Health & Human Services | National Institutes of Health (NIH)
ID : GM68933
Organisme : U.S. Department of Health & Human Services | National Institutes of Health (NIH)
ID : CA037157
Organisme : U.S. Department of Health & Human Services | National Institutes of Health (NIH)
ID : R35-122603

Informations de copyright

© 2024. The Author(s).

Références

Westerfield, J. M. & Barrera, F. N. Membrane receptor activation mechanisms and transmembrane peptide tools to elucidate them. J. Biol. Chem. 295, 1792–1814 (2020).
doi: 10.1074/jbc.REV119.009457 pubmed: 31879273
Stone, T. A. & Deber, C. M. Therapeutic design of peptide modulators of protein−protein interactions in membranes. Biochim. Biophys. Acta 1859, 577–585 (2017).
doi: 10.1016/j.bbamem.2016.08.013
Yin, H. et al. Activation of platelet αIIbβ3 by an exogenous peptide corresponding to the transmembrane domain of αIIb. J. Biol. Chem. 281, 36732–36741 (2006).
doi: 10.1074/jbc.M605877200 pubmed: 17032655
Ovchinnikov, V., Stone, T. A., Deber, C. M. & Karplus, M. Structure of the EmrE multidrug transporter and its use for inhibitor peptide design. Proc. Natl Acad. Sci. USA 115, E7932–E7941 (2018).
doi: 10.1073/pnas.1802177115 pubmed: 30082384 pmcid: 6112734
Alves, D. S. et al. A novel pH-dependent membrane peptide that binds to EphA2 and inhibits cell migration. elife 7, 36645 (2018).
doi: 10.7554/eLife.36645
Partridge, A. W., Melnyk, R. A., Yang, D., Bowie, J. U. & Deber, C. M. A transmembrane segment mimic derived from Escherichia coli diacylglycerol kinase inhibits protein activity. J. Biol. Chem. 278, 22056–22060 (2003).
doi: 10.1074/jbc.M210685200 pubmed: 12686537
He, L., Shobnam, N. & Hristova, K. Specific inhibition of a pathogenic receptor tyrosine kinase by its transmembrane domain. Biochim. Biophys. Acta 1808, 253–259 (2011).
doi: 10.1016/j.bbamem.2010.08.007 pubmed: 20713021
Tarasova, N. I. et al. Transmembrane inhibitors of P-glycoprotein, an ABC transporter. J. Med. Chem. 48, 3768–3775 (2005).
doi: 10.1021/jm049065t pubmed: 15916428
Percherancier, Y. et al. Bioluminescence resonance energy transfer reveals ligand-induced conformational changes in CXCR4 homo- and heterodimers. J. Biol. Chem. 280, 9895–9903 (2005).
doi: 10.1074/jbc.M411151200 pubmed: 15632118
Bloch, E. et al. Disrupting the transmembrane domain-mediated oligomerization of protein tyrosine phosphatase receptor J inhibits EGFR-driven cancer cell phenotypes. J. Biol. Chem. 294, 18796–18806 (2019).
doi: 10.1074/jbc.RA119.010229 pubmed: 31676686 pmcid: 6901304
Yin, H. et al. Computational design of peptides that target transmembrane helices. Science 315, 1817–1822 (2007).
doi: 10.1126/science.1136782 pubmed: 17395823
Heim, E. N. et al. Biologically active LIL proteins built with minimal chemical diversity. Proc. Natl Acad. Sci. USA 112, E4717–E4725 (2015).
doi: 10.1073/pnas.1514230112 pubmed: 26261320 pmcid: 4553812
He, L., Hoffmann, A. R., Serrano, C., Hristova, K. & Wimley, W. C. High-throughput selection of transmembrane sequences that enhance receptor tyrosine kinase activation. J. Mol. Biol. 412, 43–54 (2011).
doi: 10.1016/j.jmb.2011.07.004 pubmed: 21767549 pmcid: 3158823
Kroncke, B. M. et al. Documentation of an imperative to improve methods for predicting membrane protein stability. Biochemistry 55, 5002–5009 (2016).
doi: 10.1021/acs.biochem.6b00537 pubmed: 27564391
Weinstein, J. Y., Elazar, A. & Fleishman, S. J. A lipophilicity-based energy function for membrane-protein modelling and design. PLoS Comput. Biol. 15, e1007318 (2019).
doi: 10.1371/journal.pcbi.1007318 pubmed: 31461441 pmcid: 6736313
Alford, R. F., Fleming, P. J., Fleming, K. G. & Gray, J. J. Protein structure prediction and design in a biologically realistic implicit membrane. Biophys. J. 118, 2042–2055 (2020).
doi: 10.1016/j.bpj.2020.03.006 pubmed: 32224301 pmcid: 7175592
Mravic, M. et al. De novo designed transmembrane peptides activating the α5β1 integrin. Protein Eng. Des. Sel. 31, 181–190 (2018).
doi: 10.1093/protein/gzy014 pubmed: 29992271 pmcid: 6151875
Wang, Y. et al. Targeting trimeric transmembrane domain 5 of oncogenic latent membrane protein 1 using a computationally designed peptide. Chem. Sci. 10, 7584–7590 (2019).
doi: 10.1039/C9SC02474C pubmed: 31588309 pmcid: 6761861
Duart, G. et al. Computational design of BclxL inhibitors that target transmembrane domain interactions. Proc. Natl Acad. Sci. USA 120, e2219648120 (2023).
doi: 10.1073/pnas.2219648120 pubmed: 36881618 pmcid: 10089226
Teese, M. G. & Langosch, D. Role of GxxxG motifs in transmembrane domain interactions. Biochemistry 54, 5125–5135 (2015).
doi: 10.1021/acs.biochem.5b00495 pubmed: 26244771
Russ, W. P. & Engelman, D. M. The GxxxG motif: a framework for transmembrane helix-helix association. J. Mol. Biol. 296, 911–919 (2000).
doi: 10.1006/jmbi.1999.3489 pubmed: 10677291
Johnson, D. L. et al. Identification of a 13 amino acid peptide mimetic of erythropoietin and description of amino acids critical for the mimetic activity of EMP1. Biochemistry 37, 3699–3710 (1998).
doi: 10.1021/bi971956y pubmed: 9521688
Philo, J. S., Aoki, K. H., Arakawa, T., Narhi, L. O. & Wen, J. Dimerization of the extracellular domain of the erythropoietin (EPO) receptor by EPO: one high-affinity and one low-affinity interaction. Biochemistry 35, 1681–1691 (1996).
doi: 10.1021/bi9524272 pubmed: 8634300
Alford, R. F. et al. An integrated framework advancing membrane protein modeling and design. PLoS Comput. Biol. 11, e1004398 (2015).
doi: 10.1371/journal.pcbi.1004398 pubmed: 26325167 pmcid: 4556676
Ebie, A. Z. & Fleming, K. G. Dimerization of the erythropoietin receptor transmembrane domain in micelles. J. Mol. Biol. 366, 517–524 (2007).
doi: 10.1016/j.jmb.2006.11.035 pubmed: 17173930
Ruan, W., Becker, V., Klingmüller, U. & Langosch, D. The interface between self-assembling erythropoietin receptor transmembrane segments corresponds to a membrane-spanning leucine zipper. J. Biol. Chem. 279, 3273–3279 (2004).
doi: 10.1074/jbc.M309311200 pubmed: 14602718
Constantinescu, S. N. et al. Ligand-independent oligomerization of cell-surface erythropoietin receptor is mediated by the transmembrane domain. Proc. Natl Acad. Sci. USA 98, 4379–4384 (2001).
doi: 10.1073/pnas.081069198 pubmed: 11296286 pmcid: 31843
Kubatzky, K. F. et al. Self assembly of the transmembrane domain promotes signal transduction through the erythropoietin receptor. Curr. Biol. 11, 110–115 (2001).
doi: 10.1016/S0960-9822(01)00018-5 pubmed: 11231127
Seubert, N. et al. Active and inactive orientations of the transmembrane and cytosolic domains of the erythropoietin receptor dimer. Mol. cell 12, 1239–1250 (2003).
doi: 10.1016/S1097-2765(03)00389-7 pubmed: 14636581
Adamian, L. & Liang, J. Interhelical hydrogen bonds and spatial motifs in membrane proteins: polar clamps and serine zippers. Proteins 47, 209–218 (2002).
doi: 10.1002/prot.10071 pubmed: 11933067
North, B. et al. Characterization of a membrane protein folding motif, the Ser zipper, using designed peptides. J. Mol. Biol. 359, 930–939 (2006).
doi: 10.1016/j.jmb.2006.04.001 pubmed: 16697010
Zhang, S. Q. et al. The membrane- and soluble-protein helix−helix interactome: similar geometry via different interactions. Structure 23, 527–541 (2015).
doi: 10.1016/j.str.2015.01.009 pubmed: 25703378 pmcid: 4351763
Walters, R. F. & DeGrado, W. F. Helix-packing motifs in membrane proteins. Proc. Natl Acad. Sci. USA 103, 13658–13663 (2006).
doi: 10.1073/pnas.0605878103 pubmed: 16954199 pmcid: 1564267
Grigoryan, G. & Degrado, W. F. Probing designability via a generalized model of helical bundle geometry. J. Mol. Biol. 405, 1079–1100 (2011).
doi: 10.1016/j.jmb.2010.08.058 pubmed: 20932976
Zhang, Y., Kulp, D. W., Lear, J. D. & DeGrado, W. F. Experimental and computational evaluation of forces directing the association of transmembrane helices. J. Am. Chem. Soc. 131, 11341–11343 (2009).
doi: 10.1021/ja904625b pubmed: 19722646
Elbaz, Y., Salomon, T. & Schuldiner, S. Identification of a glycine motif required for packing in EmrE, a multidrug transporter from Escherichia coli. J. Biol. Chem. 283, 12276–12283 (2008).
doi: 10.1074/jbc.M710338200 pubmed: 18321856 pmcid: 2431008
Senes, A. et al. E(z), a depth-dependent potential for assessing the energies of insertion of amino acid side-chains into membranes: derivation and applications to determining the orientation of transmembrane and interfacial helices. J. Mol. Biol. 366, 436–448 (2007).
doi: 10.1016/j.jmb.2006.09.020 pubmed: 17174324
Fleishman, S. J. et al. RosettaScripts: a scripting language interface to the Rosetta macromolecular modeling suite. PLoS ONE 6, e20161 (2011).
doi: 10.1371/journal.pone.0020161 pubmed: 21731610 pmcid: 3123292
Sheffler, W. & Baker, D. RosettaHoles: rapid assessment of protein core packing for structure prediction, refinement, design, and validation. Protein Sci. 18, 229–239 (2009).
doi: 10.1002/pro.8 pubmed: 19177366
Lin, Z. et al. Evolutionary-scale prediction of atomic level protein structure with a language model. Science 379, 1123–1130 (2023).
doi: 10.1126/science.ade2574 pubmed: 36927031
Karabadzhak, A. G. et al. Two transmembrane dimers of the bovine papillomavirus E5 oncoprotein clamp the PDGF β receptor in an active dimeric conformation. Proc. Natl Acad. Sci. USA 114, E7262–E7271 (2017).
doi: 10.1073/pnas.1705622114 pubmed: 28808001 pmcid: 5584431
Cristian, L., Lear, J. D. & DeGrado, W. F. Determination of membrane protein stability via thermodynamic coupling of folding to thiol-disulfide interchange. Protein Sci. 12, 1732–1740 (2003).
doi: 10.1110/ps.0378603 pubmed: 12876322 pmcid: 2323959
Cristian, L., Lear, J. D. & DeGrado, W. F. Use of thiol-disulfide equilibria to measure the energetics of assembly of transmembrane helices in phospholipid bilayers. Proc. Natl Acad. Sci. USA 100, 14772–14777 (2003).
doi: 10.1073/pnas.2536751100 pubmed: 14657351 pmcid: 299801
Cabantous, S., Terwilliger, T. C. & Waldo, G. S. Protein tagging and detection with engineered self-assembling fragments of green fluorescent protein. Nat. Biotechnol. 23, 102–107 (2005).
doi: 10.1038/nbt1044 pubmed: 15580262
Choma, C., Gratkowski, H., Lear, J. D. & DeGrado, W. F. Asparagine-mediated self-association of a model transmembrane helix. Nat. Struct. Mol. Biol. 7, 161–166 (2000).
doi: 10.1038/72440
Li, M. et al. A fluorescence energy transfer method for analyzing protein oligomeric structure: application to phospholamban. Biophys. J. 76, 2587–2599 (1999).
doi: 10.1016/S0006-3495(99)77411-4 pubmed: 10233073 pmcid: 1300228
Lear, J. D., Stouffer, A. L., Gratkowski, H., Nanda, V. & Degrado, W. F. Association of a model transmembrane peptide containing Gly in a heptad sequence motif. Biophys. J. 87, 3421–3429 (2004).
doi: 10.1529/biophysj.103.032839 pubmed: 15315956 pmcid: 1304808
Lear, J. D., Gratkowski, H., Adamian, L., Liang, J. & DeGrado, W. F. Position-dependence of stabilizing polar interactions of asparagine in transmembrane helical bundles. Biochemistry 42, 6400–6407 (2003).
doi: 10.1021/bi020573j pubmed: 12767221
Federman, R. S., Boguraev, A.-S., Heim, E. N. & DiMaio, D. Biologically active ultra-simple proteins reveal principles of transmembrane domain interactions. J. Mol. Biol. 431, 3753–3770 (2019).
doi: 10.1016/j.jmb.2019.07.009 pubmed: 31301406 pmcid: 6733641
He, L. et al. Single methyl groups can act as toggle switches to specify transmembrane protein−protein interactions. eLife 6, 27701 (2017).
doi: 10.7554/eLife.27701
He, L. et al. Transmembrane protein aptamer induces cooperative signaling by the epo receptor and the cytokine receptor β-common subunit. iScience 17, 167–181 (2019).
doi: 10.1016/j.isci.2019.06.027 pubmed: 31279934 pmcid: 6614117
Litvinov, R. I. et al. Unique transmembrane domain interactions differentially modulate integrin αvβ3 and αIIbβ3 function. Proc. Natl Acad. Sci. USA 116, 12295–12300 (2019).
doi: 10.1073/pnas.1904867116 pubmed: 31160446 pmcid: 6589676
Livnah, O. et al. An antagonist peptide–EPO receptor complex suggests that receptor dimerization is not sufficient for activation. Nat. Struct. Mol. Biol. 5, 993–1004 (1998).
doi: 10.1038/2965
Livnah, O. et al. Functional mimicry of a protein hormone by a peptide agonist: the EPO receptor complex at 2.8 A. Science 273, 464–471 (1996).
doi: 10.1126/science.273.5274.464 pubmed: 8662530
Syed, R. S. et al. Efficiency of signalling through cytokine receptors depends critically on receptor orientation. Nature 395, 511–516 (1998).
doi: 10.1038/26773 pubmed: 9774108
Zhang, H., Wilson, I. A. & Lerner, R. A. Selection of antibodies that regulate phenotype from intracellular combinatorial antibody libraries. Proc. Natl Acad. Sci. USA 109, 15728–15733 (2012).
doi: 10.1073/pnas.1214275109 pubmed: 23019357 pmcid: 3465454
Mohan, K. et al. Topological control of cytokine receptor signaling induces differential effects in hematopoiesis. Science 364, eaav7532 (2019).
doi: 10.1126/science.aav7532 pubmed: 31123111 pmcid: 7274355
Lee, W., Tonelli, M. & Markley, J. L. NMRFAM-SPARKY: enhanced software for biomolecular NMR spectroscopy. Bioinformatics 31, 1325–1327 (2015).
doi: 10.1093/bioinformatics/btu830 pubmed: 25505092
Delaglio, F. et al. NMRPipe: a multidimensional spectral processing system based on UNIX pipes. J. Biomol. NMR 6, 277–293 (1995).
doi: 10.1007/BF00197809 pubmed: 8520220
Dang, B. et al. SNAC-tag for sequence-specific chemical protein cleavage. Nat. Methods 16, 319–322 (2019).
doi: 10.1038/s41592-019-0357-3 pubmed: 30923372 pmcid: 6443254

Auteurs

Marco Mravic (M)

Department of Pharmaceutical Chemistry, School of Pharmacy, University of California, San Francisco, CA, USA. mmravic@scripps.edu.
Department of Integrative Structural and Computational Biology, The Scripps Research Institute, La Jolla, CA, USA. mmravic@scripps.edu.

Li He (L)

Department of Genetics, Yale School of Medicine, New Haven, CT, USA.

Huong T Kratochvil (HT)

Department of Pharmaceutical Chemistry, School of Pharmacy, University of California, San Francisco, CA, USA.
Department of Chemistry, University of North Carolina Chapel Hill, Chapel Hill, NC, USA.

Hailin Hu (H)

Department of Pharmaceutical Chemistry, School of Pharmacy, University of California, San Francisco, CA, USA.
School of Medicine, Tsinghua University, Beijing, China.

Sarah E Nick (SE)

Department of Pharmaceutical Chemistry, School of Pharmacy, University of California, San Francisco, CA, USA.

Weiya Bai (W)

Department of Genetics, Yale School of Medicine, New Haven, CT, USA.

Anne Edwards (A)

Department of Genetics, Yale School of Medicine, New Haven, CT, USA.

Hyunil Jo (H)

Department of Pharmaceutical Chemistry, School of Pharmacy, University of California, San Francisco, CA, USA.

Yibing Wu (Y)

Department of Pharmaceutical Chemistry, School of Pharmacy, University of California, San Francisco, CA, USA.

Daniel DiMaio (D)

Department of Genetics, Yale School of Medicine, New Haven, CT, USA. daniel.dimaio@yale.edu.
Department of Therapeutic Radiology, Yale School of Medicine, New Haven, CT, USA. daniel.dimaio@yale.edu.
Department of Molecular Biophysics & Biochemistry, Yale University, New Haven, CT, USA. daniel.dimaio@yale.edu.
Yale Cancer Center, New Haven, CT, USA. daniel.dimaio@yale.edu.

William F DeGrado (WF)

Department of Pharmaceutical Chemistry, School of Pharmacy, University of California, San Francisco, CA, USA. bill.degrado@ucsf.edu.

Classifications MeSH