A hybrid approach reveals the allosteric regulation of GTP cyclohydrolase I.


Journal

Proceedings of the National Academy of Sciences of the United States of America
ISSN: 1091-6490
Titre abrégé: Proc Natl Acad Sci U S A
Pays: United States
ID NLM: 7505876

Informations de publication

Date de publication:
15 12 2020
Historique:
pubmed: 25 11 2020
medline: 2 2 2021
entrez: 24 11 2020
Statut: ppublish

Résumé

Guanosine triphosphate (GTP) cyclohydrolase I (GCH1) catalyzes the conversion of GTP to dihydroneopterin triphosphate (H2NTP), the initiating step in the biosynthesis of tetrahydrobiopterin (BH4). Besides other roles, BH4 functions as cofactor in neurotransmitter biosynthesis. The BH4 biosynthetic pathway and GCH1 have been identified as promising targets to treat pain disorders in patients. The function of mammalian GCH1s is regulated by a metabolic sensing mechanism involving a regulator protein, GCH1 feedback regulatory protein (GFRP). GFRP binds to GCH1 to form inhibited or activated complexes dependent on availability of cofactor ligands, BH4 and phenylalanine, respectively. We determined high-resolution structures of human GCH1-GFRP complexes by cryoelectron microscopy (cryo-EM). Cryo-EM revealed structural flexibility of specific and relevant surface lining loops, which previously was not detected by X-ray crystallography due to crystal packing effects. Further, we studied allosteric regulation of isolated GCH1 by X-ray crystallography. Using the combined structural information, we are able to obtain a comprehensive picture of the mechanism of allosteric regulation. Local rearrangements in the allosteric pocket upon BH4 binding result in drastic changes in the quaternary structure of the enzyme, leading to a more compact, tense form of the inhibited protein, and translocate to the active site, leading to an open, more flexible structure of its surroundings. Inhibition of the enzymatic activity is not a result of hindrance of substrate binding, but rather a consequence of accelerated substrate binding kinetics as shown by saturation transfer difference NMR (STD-NMR) and site-directed mutagenesis. We propose a dissociation rate controlled mechanism of allosteric, noncompetitive inhibition.

Identifiants

pubmed: 33229582
pii: 2013473117
doi: 10.1073/pnas.2013473117
pmc: PMC7750480
doi:

Substances chimiques

GCHFR protein, human 0
Intracellular Signaling Peptides and Proteins 0
Biopterins 0
Phenylalanine 47E5O17Y3R
GCH1 protein, human EC 3.5.4.16
GTP Cyclohydrolase EC 3.5.4.16
sapropterin EGX657432I

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

31838-31849

Informations de copyright

Copyright © 2020 the Author(s). Published by PNAS.

Déclaration de conflit d'intérêts

Competing interest statement: R.E., R.M., M.R., D.R., M.B., L.W., M.Z., and H.N. were employees of Boehringer Ingelheim at the time of this work.

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Auteurs

Rebecca Ebenhoch (R)

Medicinal Chemistry, Boehringer Ingelheim Pharma GmbH & Co. KG, 88397 Biberach an der Riss, Germany.

Simone Prinz (S)

Structural Biology, Max Planck Institute of Biophysics, 60438 Frankfurt am Main, Germany.

Susann Kaltwasser (S)

Structural Biology, Max Planck Institute of Biophysics, 60438 Frankfurt am Main, Germany.

Deryck J Mills (DJ)

Structural Biology, Max Planck Institute of Biophysics, 60438 Frankfurt am Main, Germany.

Robert Meinecke (R)

Medicinal Chemistry, Boehringer Ingelheim Pharma GmbH & Co. KG, 88397 Biberach an der Riss, Germany.

Martin Rübbelke (M)

Medicinal Chemistry, Boehringer Ingelheim Pharma GmbH & Co. KG, 88397 Biberach an der Riss, Germany.

Dirk Reinert (D)

Medicinal Chemistry, Boehringer Ingelheim Pharma GmbH & Co. KG, 88397 Biberach an der Riss, Germany.

Margit Bauer (M)

Medicinal Chemistry, Boehringer Ingelheim Pharma GmbH & Co. KG, 88397 Biberach an der Riss, Germany.

Lisa Weixler (L)

Medicinal Chemistry, Boehringer Ingelheim Pharma GmbH & Co. KG, 88397 Biberach an der Riss, Germany.

Markus Zeeb (M)

Medicinal Chemistry, Boehringer Ingelheim Pharma GmbH & Co. KG, 88397 Biberach an der Riss, Germany.

Janet Vonck (J)

Structural Biology, Max Planck Institute of Biophysics, 60438 Frankfurt am Main, Germany.

Herbert Nar (H)

Medicinal Chemistry, Boehringer Ingelheim Pharma GmbH & Co. KG, 88397 Biberach an der Riss, Germany; herbert.nar@boehringer-ingelheim.com.

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Classifications MeSH