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Title: Distinct regions that control ion selectivity and calcium-dependent activation in the bestrophin ion channel

Journal Article · · Proceedings of the National Academy of Sciences of the United States of America
 [1];  [1];  [1]
  1. Memorial Sloan Kettering Cancer Center, New York, NY (United States)

Cytoplasmic calcium (Ca2+) activates the bestrophin anion channel, allowing chloride ions to flow down their electrochemical gradient. Mutations in bestrophin 1 (BEST1) cause macular degenerative disorders. Previously, we determined an X-ray structure of chicken BEST1 that revealed the architecture of the channel. Here, we present electrophysiological studies of purified wild-type and mutant BEST1 channels and an X-ray structure of a Ca2+-independent mutant. From these experiments, we identify regions of BEST1 responsible for Ca2+ activation and ion selectivity. A “Ca2+ clasp” within the channel’s intracellular region acts as a sensor of cytoplasmic Ca2+. Alanine substitutions within a hydrophobic “neck” of the pore, which widen it, cause the channel to be constitutively active, irrespective of Ca2+. We conclude that the primary function of the neck is as a “gate” that controls chloride permeation in a Ca2+-dependent manner. In contrast to what others have proposed, we find that the neck is not a major contributor to the channel’s ion selectivity. We find that mutation of a cytosolic “aperture” of the pore does not perturb the Ca2+ dependence of the channel or its preference for anions over cations, but its mutation dramatically alters relative permeabilities among anions. The data suggest that the aperture functions as a size-selective filter that permits the passage of small entities such as partially dehydrated chloride ions while excluding larger molecules such as amino acids. Furthermore, unlike ion channels that have a single “selectivity filter,” in bestrophin, distinct regions of the pore govern anion-vs.-cation selectivity and the relative permeabilities among anions.

Research Organization:
Argonne National Laboratory (ANL), Argonne, IL (United States)
Sponsoring Organization:
USDOE; National Institutes of Health (NIH); Memorial Sloan Kettering Cancer Center
Grant/Contract Number:
AC02-06CH11357; ACB-12002; AGM-12006; P41 GM103403; S10 RR029205; R01 GM110396; P30 CA008748
OSTI ID:
1335989
Journal Information:
Proceedings of the National Academy of Sciences of the United States of America, Vol. 113, Issue 47; ISSN 0027-8424
Publisher:
National Academy of SciencesCopyright Statement
Country of Publication:
United States
Language:
ENGLISH
Citation Metrics:
Cited by: 46 works
Citation information provided by
Web of Science

References (47)

Bestrophins and retinopathies journal March 2010
Regulation of Bestrophin Cl Channels by Calcium: Role of the C Terminus journal November 2008
Linking Crystallographic Model and Data Quality journal May 2012
Principles of activation and permeation in an anion-selective Cys-loop receptor journal May 2011
Bestrophin 1 is indispensable for volume regulation in human retinal pigment epithelium cells journal May 2015
Hydrophobic Gating in Ion Channels journal January 2015
HKL -3000: the integration of data reduction and structure solution – from diffraction images to an initial model in minutes journal July 2006
HOLE: A program for the analysis of the pore dimensions of ion channel structural models journal December 1996
Ca 2+ -activated Cl Current from Human Bestrophin-4 in Excised Membrane Patches journal May 2006
Calcium-Activated Chloride Channels journal March 2005
Crystal structures of a double-barrelled fluoride ion channel journal September 2015
A Practical Guide to the Preparation of Ca2+ Buffers book January 1994
Features and development of Coot journal March 2010
Two Bestrophins Cloned from Xenopus laevis Oocytes Express Ca 2+ -activated Cl - Currents journal August 2003
A hydrophobic barrier deep within the inner pore of the TWIK-1 K2P potassium channel journal July 2014
Rescue of Volume-regulated Anion Current by Bestrophin Mutants with Altered Charge Selectivity journal October 2008
A Survey of Aspartate−Phenylalanine and Glutamate−Phenylalanine Interactions in the Protein Data Bank: Searching for Anion−π Pairs journal April 2011
PHENIX: a comprehensive Python-based system for macromolecular structure solution journal January 2010
Anion-Sensitive Fluorophore Identifies the Drosophila Swell-Activated Chloride Channel in a Genome-Wide RNA Interference Screen journal October 2012
Missense Mutations in a Retinal Pigment Epithelium Protein, Bestrophin-1, Cause Retinitis Pigmentosa journal November 2009
The Anion-Selective Pore of the Bestrophins, a Family of Chloride Channels Associated with Retinal Degeneration journal May 2006
Version 1.2 of the Crystallography and NMR system journal October 2007
Molecular Physiology of Bestrophins: Multifunctional Membrane Proteins Linked to Best Disease and Other Retinopathies journal April 2008
A Preference for Edgewise Interactions between Aromatic Rings and Carboxylate Anions:  The Biological Relevance of Anion−Quadrupole Interactions journal July 2007
Disease-associated missense mutations in bestrophin-1 affect cellular trafficking and anion conductance journal August 2011
Characterization of the effects of Cl− channel modulators on TMEM16A and bestrophin-1 Ca2+ activated Cl− channels journal August 2014
Anion Permeation in Ca2+-Activated Cl− Channels journal December 2000
Determinants of Anion Permeation in the Second Transmembrane Domain of the Mouse Bestrophin-2 Chloride Channel journal September 2004
Structure and insights into the function of a Ca2+-activated Cl− channel journal October 2014
Volume sensitivity of the bestrophin family of chloride channels: Volume sensitivity of bestrophin currents journal January 2005
The Structure of an Open Form of an E. coli Mechanosensitive Channel at 3.45 A Resolution journal August 2008
Autosomal Recessive Vitelliform Macular Dystrophy in a Large Cohort of Vitelliform Macular Dystrophy Patients journal January 2011
Structure and selectivity in bestrophin ion channels journal September 2014
The Structure of the Potassium Channel: Molecular Basis of K+ Conduction and Selectivity journal April 1998
Mouse Bestrophin-2 Is a Bona fide Cl Channel : Identification of a Residue Important in Anion Binding and Conduction journal March 2004
The vitelliform macular dystrophy protein defines a new family of chloride channels journal March 2002
Identification of the gene responsible for Best macular dystrophy journal July 1998
Mutations in a novel gene, VMD2, encoding a protein of unknown properties cause juvenile-onset vitelliform macular dystrophy (Best's disease) journal September 1998
Calcium-Activated Chloride Channels book January 2010
Structure and Selectivity in Bestrophin Ion Channels journal January 2015
PARP1 exhibits enhanced association and catalytic efficiency with γH2A.X-nucleosome journal December 2019
Cavin1 intrinsically disordered domains are essential for fuzzy electrostatic interactions and caveola formation journal February 2021
The oligomeric structures of plant cryptochromes journal May 2020
PHENIX: a comprehensive Python-based system for macromolecular structure solution. text January 2010
Disease-associated missense mutations in bestrophin-1 affect cellular trafficking and anion conductance text January 2011
Structure-Function Analysis of the Bestrophin Family of Anion Channels journal August 2003
Ca2+-activated Chloride Channels Go Molecular journal March 2004

Cited By (19)

GABAergic‐astrocyte signaling: A refinement of inhibitory brain networks journal May 2019
The lysosomal potassium channel TMEM175 adopts a novel tetrameric architecture journal July 2017
ATP activates bestrophin ion channels through direct interaction journal August 2018
Next generation sequencing identifies novel disease-associated BEST1 mutations in Bestrophinopathy patients journal July 2018
Dual Ca2+-dependent gates in human Bestrophin1 underlie disease-causing mechanisms of gain-of-function mutations journal June 2019
Water and hydrophobic gates in ion channels and nanopores journal January 2018
BEST1 gene therapy corrects a diffuse retina-wide microdetachment modulated by light exposure journal March 2018
An allosteric mechanism of inactivation in the calcium-dependent chloride channel BEST1 journal September 2018
A clearer image of the structure and regulation of bestrophin journal October 2018
Human iPSC modeling reveals mutation-specific responses to gene therapy in Best disease posted_content January 2020
Pathogenicity of new BEST1 variants identified in Italian patients with best vitelliform macular dystrophy assessed by computational structural biology journal October 2019
Novel Missense Mutations in BEST1 Are Associated with Bestrophinopathies in Lebanese Patients journal February 2019
Chloride Channels in Astrocytes: Structure, Roles in Brain Homeostasis and Implications in Disease journal February 2019
Structural basis for ion selectivity in TMEM175 K+ channels text January 2020
Dual Ca2+-Dependent Gates in Human Bestrophin1 Underlie Disease-Causing Mechanisms of Gain-Of-Function Mutations journal February 2020
Patient-Specific Mutations Impair Bestrophin1's Essential Role in Mediating Ca2+-Dependent CL- Currents in Human RPE journal February 2018
Structural basis for ion selectivity in TMEM175 K+ channels journal April 2020
A BEST example of channel structure annotation by molecular simulation journal March 2017
Astrocyte, a Promising Target for Mood Disorder Interventions journal June 2019