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Items: 17

1.

Oral Migalastat HCl Leads to Greater Systemic Exposure and Tissue Levels of Active α-Galactosidase A in Fabry Patients when Co-Administered with Infused Agalsidase.

Warnock DG, Bichet DG, Holida M, Goker-Alpan O, Nicholls K, Thomas M, Eyskens F, Shankar S, Adera M, Sitaraman S, Khanna R, Flanagan JJ, Wustman BA, Barth J, Barlow C, Valenzano KJ, Lockhart DJ, Boudes P, Johnson FK.

PLoS One. 2015 Aug 7;10(8):e0134341. doi: 10.1371/journal.pone.0134341. eCollection 2015.

2.

Evidence that small molecule enhancement of β-hexosaminidase activity corrects the behavioral phenotype in Dutch APP(E693Q) mice through reduction of ganglioside-bound Aβ.

Knight EM, Williams HN, Stevens AC, Kim SH, Kottwitz JC, Morant AD, Steele JW, Klein WL, Yanagisawa K, Boyd RE, Lockhart DJ, Sjoberg ER, Ehrlich ME, Wustman BA, Gandy S.

Mol Psychiatry. 2015 Feb;20(1):109-17. doi: 10.1038/mp.2014.135. Epub 2014 Oct 28.

3.

A GCase chaperone improves motor function in a mouse model of synucleinopathy.

Richter F, Fleming SM, Watson M, Lemesre V, Pellegrino L, Ranes B, Zhu C, Mortazavi F, Mulligan CK, Sioshansi PC, Hean S, De La Rosa K, Khanna R, Flanagan J, Lockhart DJ, Wustman BA, Clark SW, Chesselet MF.

Neurotherapeutics. 2014 Oct;11(4):840-56. doi: 10.1007/s13311-014-0294-x.

4.

The pharmacological chaperone AT2220 increases the specific activity and lysosomal delivery of mutant acid alpha-glucosidase, and promotes glycogen reduction in a transgenic mouse model of Pompe disease.

Khanna R, Powe AC Jr, Lun Y, Soska R, Feng J, Dhulipala R, Frascella M, Garcia A, Pellegrino LJ, Xu S, Brignol N, Toth MJ, Do HV, Lockhart DJ, Wustman BA, Valenzano KJ.

PLoS One. 2014 Jul 18;9(7):e102092. doi: 10.1371/journal.pone.0102092. eCollection 2014.

5.

Pharmacological chaperones as therapeutics for lysosomal storage diseases.

Boyd RE, Lee G, Rybczynski P, Benjamin ER, Khanna R, Wustman BA, Valenzano KJ.

J Med Chem. 2013 Apr 11;56(7):2705-25. doi: 10.1021/jm301557k. Epub 2013 Mar 11. Review.

PMID:
23363020
6.

Lysosomal dysfunction in a mouse model of Sandhoff disease leads to accumulation of ganglioside-bound amyloid-β peptide.

Keilani S, Lun Y, Stevens AC, Williams HN, Sjoberg ER, Khanna R, Valenzano KJ, Checler F, Buxbaum JD, Yanagisawa K, Lockhart DJ, Wustman BA, Gandy S.

J Neurosci. 2012 Apr 11;32(15):5223-36. doi: 10.1523/JNEUROSCI.4860-11.2012.

7.

The pharmacological chaperone isofagomine increases the activity of the Gaucher disease L444P mutant form of beta-glucosidase.

Khanna R, Benjamin ER, Pellegrino L, Schilling A, Rigat BA, Soska R, Nafar H, Ranes BE, Feng J, Lun Y, Powe AC, Palling DJ, Wustman BA, Schiffmann R, Mahuran DJ, Lockhart DJ, Valenzano KJ.

FEBS J. 2010 Apr;277(7):1618-38. doi: 10.1111/j.1742-4658.2010.07588.x. Epub 2010 Feb 10.

8.

Cellular and tissue localization of globotriaosylceramide in Fabry disease.

Askari H, Kaneski CR, Semino-Mora C, Desai P, Ang A, Kleiner DE, Perlee LT, Quezado M, Spollen LE, Wustman BA, Schiffmann R.

Virchows Arch. 2007 Oct;451(4):823-34. Epub 2007 Aug 3.

PMID:
17674039
9.

Structure of acid beta-glucosidase with pharmacological chaperone provides insight into Gaucher disease.

Lieberman RL, Wustman BA, Huertas P, Powe AC Jr, Pine CW, Khanna R, Schlossmacher MG, Ringe D, Petsko GA.

Nat Chem Biol. 2007 Feb;3(2):101-7. Epub 2006 Dec 24.

PMID:
17187079
11.

Characterization of two molluscan crystal-modulating biomineralization proteins and identification of putative mineral binding domains.

Michenfelder M, Fu G, Lawrence C, Weaver JC, Wustman BA, Taranto L, Evans JS, Morse DE.

Biopolymers. 2003 Dec;70(4):522-33. Erratum in: Biopolymers. 2004 Feb 5;73(2):291.

PMID:
14648763
12.

Structure-function studies of the lustrin A polyelectrolyte domains, RKSY and D4.

Wustman BA, Weaver JC, Morse DE, Evans JS.

Connect Tissue Res. 2003;44 Suppl 1:10-5.

PMID:
12952167
13.
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15.

Effects of elevated CO2 and O3 on aspen clones varying in O3 sensitivity: can CO2 ameliorate the harmful effects of O3?

Wustman BA, Oksanen E, Karnosky DF, Noormets A, Isebrands JG, Pregitzer KS, Hendrey GR, Sober J, Podila GK.

Environ Pollut. 2001;115(3):473-81.

PMID:
11789927

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