Journal of the American Chemical Society
Page 12 of 12
1
2
3
4
2013, 92, 245–251.
(8) Martin, E., Schüle, R., Smets, K., Rastetter, A., Bou-
5
6
7
8
ASSOCIATED CONTENT
khris, A., Loureiro, J. L., Gonzalez, M. A., Mundwiller, E.,
Deconinck, T., Wessner, M., Jornea, L., Oteyza, A. C.,
Durr, A., Martin, J. J., Schöls, L., Mhiri, C., Lamari, F.,
Züchner, S., De Jonghe, P., Kabashi, E., Brice, A., Stevan-
in, G. Am. J. Hum. Genet. 2013, 92, 238–244.
(9) Sultana, S., Truong, N. Y., Vieira, D. B., Wigger, J. G.
D., Forrester, A. M., Veinotte, C. J., Berman, J. N., van der
Spoel, A. C. Zebrafish 2016, 13, 177–187.
(10) Marques, A. R. A., Aten, J., Ottenhoff, R., Van
Roomen, C. P. A. A., Moro, D. H., Claessen, N., Veloz, M.
F. V., Zhou, K., Lin, Z., Mirzaian, M., Boot, R. G., De
Zeeuw, C. I., Overkleeft, H. S., Yildiz, Y., Aerts, J. M. F.
G. PLoS One 2015, 10, 1–18.
(11) Overkleeft, H. S., Renkema, G. H., Neele, J., Vianel-
lo, P., Hung, I. O., Strijland, A., van der Burg, A. M.,
Koomen, G., Pandit, U. K., Aerts, J. M. F. G. J. Biol.
Chem. 1998, 273, 26522–26527.
(12) Wennekes, T., van den Berg, R. J. B. H. N., Boot, R.
G., van der Marel, G. A., Overkleeft, H. S., Aerts, J. M. F.
G. Angew. Chem. Int. Ed. 2009, 48, 8848–8869.
(13) Kallemeijn, W. W., Li, K.-Y., Witte, M. D., Marques,
A. R. A., Aten, J., Scheij, S., Jiang, J., Willems, L. I.,
Voorn-Brouwer, T. M., van Roomen, C. P. A. A., Otten-
hoff, R., Boot, R. G., van den Elst, H., Walvoort, M. T. C.,
Florea, B. I., Codée, J. D. C., van der Marel, G. A., Aerts,
J. M. F. G., Overkleeft, H. S. Angew. Chem. Int. Ed. 2012,
124, 12697–12701.
(14) Charoenwattanasatien, R., Pengthaisong, S., Breen, I.,
Mutoh, R., Sansenya, S., Hua, Y., Tankrathok, A., Wu, L.,
Songsiriritthigul, C., Tanaka, H., Williams, S. J., Davies,
G. J., Kurisu, G., Cairns, J. R. K. ACS Chem. Biol. 2016,
11, 1891–1900.
Supporting Information.
Synthetic procedures for ABP 4 and (unpublished) deox-
ynojirimycin analogues, H and 13C NMR spectra, IR and
1
9
LC-MS data; Figures validating the FluoPol-ABPP method
and additional figures for the screen showing a preference
for gluco-and ido- configured iminosugars; Compound
structures and inhibitory potencies on GCS, GBA1 and
GBA2. This material is free of available free of charge via
the ACS Publications website.
10
11
12
13
14
15
16
17
18
19
20
21
22
23
24
25
26
27
28
29
30
31
32
33
34
35
36
37
38
39
40
41
42
43
44
45
46
47
48
49
50
51
52
53
54
55
56
57
58
59
60
AUTHOR INFORMATION
Corresponding Authors
*j.m.aerts@chem.leidenuniv.nl;
Author Contributions
#DL and BL contributed equally to this work
ACKNOWLEDGMENT
We thank the Netherlands Organization for Scientific Re-
search (NWO-CW, TOP-PUNT grant to HSO), the China
Scholarship Council (CSC, PhD fellowship to BL) and the
European Research Council (ERC-2011-AdG-290836
“Chembiosphing”, to HSO, ERC-2012-AdG-32294 “Gly-
copoise”, to GJD; GJD holds the Royal Society Ken Mur-
ray Research Professorship) for financial support.
REFERENCES
(1) Van Weely, S., Brandsma, M., Strijland, A., Tager, J.
M., Aerts, J. M. F. G. Biochim. Biophys. Acta 1993, 1181,
55–62.
(2) Yildiz, Y., Matern, H., Thompson, B., Allegood, J. C.,
Warren, R. L., Ramirez, D. M. O., Hammer, R. E., Hamra,
F. K., Matern, S., Russell, D. W. J. Clin. Invest. 2006, 116,
2985–2994.
(3) Boot, R. G., Verhoek, M., Donker-Koopman, W., Strij-
land, A., Van Marle, J., Overkleeft, H. S., Wennekes, T.,
Aerts, J. M. F. G. J. Biol. Chem. 2007, 282, 1305–1312.
(4) Körschen, H. G., Yildiz, Y., Raju, D. N., Schonauer,
S., Bönigk, W., Jansen, V., Kremmer, E., Kaupp, U. B.,
Wachten, D. J. Biol. Chem. 2013, 288, 3381–3393.
(5) Ferraz, M. J., Kallemeijn, W. W., Mirzaian, M., Her-
rera Moro, D., Marques, A., Wisse, P., Boot, R. G., Wil-
lems, L. I., Overkleeft, H. S., Aerts, J. M. F. G.
Biochim. Biophys. Acta 2014, 1841, 811–825.
(6) Nagata, M., Izumi, Y., Ishikawa, E., Kiyotake, R., Doi,
R., Iwai, S., Zakaria, O., Yamaji, T., Miyamoto, T., Bamba,
T., Yamasaki, S. Proc. Natl. Acad. Sci. U.S.A. 2017, 114,
3285–3294.
(7) Hammer, M. B., Eleuch-Fayache, G., Schottlaender, L.
V., Nehdi, H., Gibbs, J. R., Arepalli, S. K., Chong, S. B.,
Hernandez, D. G., Sailer, A., Liu, G., Mistry, P. K., Cai,
H., Shrader, G., Sassi, C., Bouhlal, Y., Houlden, H., Henta-
ti, F., Amouri, R., Singleton, A. B. Am. J. Hum. Genet.
(15) Bachovchin, D. A., Brown, S. J., Rosen, H., Cravatt,
B. F. Nat. Biotechnol. 2009, 27, 387–394.
(16) Wennekes, T., Meijer, A. J., Groen, A. K., Boot, R.
G., Groener, J. E., Eijk, M. Van, Ottenhoff, R., Bijl, N.,
Ghauharali, K., Song, H., Shea, T. J. O., Liu, H., Yew, N.,
Copeland, D., van den Berg, R. J., van der Marel, G. A.
Overkleeft, H. S., Aerts, J. M. F. G. J. Med. Chem. 2010,
53, 689–698.
(17) Lea, W.A., Simeonov, A. Expert Opin. Drug Discov.,
2011, 6, 17-32.
(18) Niphakis, M. J., Cravatt, B. F. Annu Rev. Biochem.
2014, 83, 341–377.
(19) Dekker, N., Voorn-Brouwer, T., Verhoek, M., Wen-
nekes, T., Narayan, R. S., Speijer, D., Hollak, C. E. M.,
Overkleeft, H. S., Boot, R. G., Aerts, J. M. F. G. Blood Cell
Mol. Dis., 2011, 46, 19–26.
(20) Bligh, E.G., Dyer, W.J. Can. J. Biochem. Phys., 1959,
37, 911–917.
ACS Paragon Plus Environment