Nuclear Magnetic Resonance Solution Conformation of -Conotoxin AuIB, an 3 4 Subtype-selective Neuronal Nicotinic Acetylcholine Receptor Antagonist* (Received for publication, October 4, 1999, and in revised form, December 17, 1999) Jee-Hyun Cho‡, K. Hun Mok‡, Baldomero M. Olivera§, J. Michael McIntosh§, Kyu-Hwan Park‡, and Kyou-Hoon Han‡ From the Protein Engineering Laboratory, Korea Research Institute of Bioscience and Biotechnology, Yusong, Taejon 305- 600, Republic of Korea and Departments of §Biology and Psychiatry, University of Utah, Salt Lake City, Utah 84112 The neuronal nicotinic acetylcholine receptors consti- tute a highly diverse group, with subtypes consisting of pentameric combinations of and subunits. -Cono- toxins are a homologous series of small peptides that antagonize these receptors. We present the three-dimen- sional solution structure of -conotoxin AuIB, the first 15-residue -conotoxin known to selectively block the 3 4 nicotinic acetylcholine receptor subtype. The pair- wise backbone and heavy-atom root mean square devi- ation for an ensemble of 20 structures are 0.269 and 0.720 Å, respectively. The overall fold of -conotoxin AuIB closely resembles that of the 4/7 subfamily -conotox- ins. However, the absence of Tyr 15 , normally present in other 4/7 members, results in tight bending of the back- bone at the C terminus and effectively renders Asp 14 to assume the spatial location of Tyr 15 present in other neuronal 4/7 -conotoxins. Structural comparison of -conotoxin AuIB with the 3 2 subtype-specific -cono- toxin MII shows different electrostatic surface charge distributions, which may be important in differential receptor subtype recognition. The -conotoxins are small neuropharmacologically active peptides of Conus origin that antagonize the nicotinic acetyl- choline receptor (nAChR) 1 (1). The natural diversity of biosyn- thesized conotoxin peptides has led to the classification of a wide spectrum of disulfide-bridged peptides, which attack var- ious ligand and ion-gated channels and receptors (1). The nic- otinic acetylcholine receptors exhibit considerable diversity in their own right because of the different compositions found in the pentameric subunits constituting each nAChR subtype (2). Although the mammalian neuromuscular subtype comprises ( 1 ) 2  or ( 1 ) 2  subunits, the neuronal subtypes are com- paratively more diverse with their hetero- or homopentameric combinations of ( 2 9 ) or ( 2 4 ) subunits (3, 4). The general conotoxin strategy of diversification is “combinatorial” (5), in which amino acid residues are varied within a given disulfide framework to specifically and selectively bind various subtypes of the target channel or receptor. For the case of -conotoxins, target selectivity is essentially defined depending on which subunit interface of the nAChR (e.g. 1 /, 1 /, and 3 / 2 ) each individual -conotoxin preferentially binds to (1). Highly selective -conotoxins that permit differential blocking of diverse nAChR subtypes have served as effective tools in studying these receptors (1). Of recent particular interest are the -conotoxins that act on neuronal nAChRs. For example, -conotoxin AuIB specifically targets the 3 4 subtype (6), whereas -conotoxin MII selec- tively blocks the 3 2 subtype (7). On the other hand, -cono- toxin ImI, the smallest of all -conotoxins and distinct because of its 4/3 disulfide framework 2 , is a specific antagonist of the homomeric 7 subtype (8). In addition, other neuronal -cono- toxins such as PnIA (9), PnIB (9), and EpI (10) that are less selective to a particular nAChR subtype have also been iden- tified (Table I). As shown in Table I, most neuronal -conotoxins belong to the 4/7 subfamily, in which the amino acid sequence varies within disulfide bridge-enclosed loops of 4 and 7 residues. In- terestingly, -conotoxin EI, unique in its specificity for the 1 / subunit interface in Torpedo neuromuscular nAChR, belongs to the same 4/7 subfamily (11). The recent increase in the un- derstanding of these conotoxins has been attributable to the advances in the structural characterization of these molecules. High-resolution three-dimensional structures of 4/7 subfam- ily -conotoxins PnIA (12), PnIB (13), MII (14, 15), and [Tyr 15 ]EpI (16) show that, when backbone-superimposed, their backbone fold is extremely similar. The recently solved NMR structure of -conotoxin EI also has the same overall molecular fold as the 4/7 neuronal -conotoxins 3 despite the variation in its sequence both within the disulfide loops and at the N ter- minus (Table I). These observations reaffirm that conotoxins are based on common three-dimensional scaffolds and that their subtype selectivity is conferred through sequence varia- tion of a selected number of residues (5). Through structural elucidation of these highly selective -conotoxins and their analogs (17, 18), we have been using the reverse-mapping approach first to identify receptor subtype specific determinants in the ligands and second to indirectly probe the regions in nAChR responsible for binding agonists * This research was supported in part by Grant NB0980 from the Ministry of Science and Technology, Korea. The costs of publication of this article were defrayed in part by the payment of page charges. This article must therefore be hereby marked “advertisement” in accordance with 18 U.S.C. Section 1734 solely to indicate this fact. The atomic coordinates and structure factors (code 1DG2) have been deposited in the Protein Data Bank, Research Collaboratory for Struc- tural Bioinformatics, Rutgers University, New Brunswick, NJ (http:wwwresb.org1). To whom correspondence should be addressed: Protein Engineering Laboratory, Korea Research Institute of Bioscience and Biotechnology, Yusong P.O. Box 115, Taejon 305-600, Republic of Korea. Tel.: 82-42- 860-4250; Fax: 82-42-860-4259; E-mail: khhan@biotech5.kribb.re.kr. 1 The abbreviations used are: nAChR, nicotinic acetylcholine recep- tor; NOE, nuclear Overhauser effect; NOESY, nuclear Overhauser ef- fect spectroscopy; COSY, correlation spectroscopy; RMSD, root mean square deviation. 2 The -conotoxins are additionally grouped according to the number of amino acid residues enclosed within each disulfide loop. Loop sizes of four residues in the first loop and seven in the second are denoted as 4/7. Other subfamilies include 3/5 and 4/3. 3 K.-H. Park, J. E. Suk, R. Jacobsen, W. R. Gray, J. M. McIntosh, B. M. Olivera, and K. Han, manuscript in preparation. THE JOURNAL OF BIOLOGICAL CHEMISTRY Vol. 275, No. 12, Issue of March 24, pp. 8680 –8685, 2000 © 2000 by The American Society for Biochemistry and Molecular Biology, Inc. Printed in U.S.A. This paper is available on line at http://www.jbc.org 8680 by guest on June 6, 2020 http://www.jbc.org/ Downloaded from