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Items: 1 to 20 of 102

1.

Modification of aspartoacylase for potential use in enzyme replacement therapy for the treatment of Canavan disease.

Zano S, Malik R, Szucs S, Matalon R, Viola RE.

Mol Genet Metab. 2011 Feb;102(2):176-80. doi: 10.1016/j.ymgme.2010.10.012. Epub 2010 Oct 30.

2.

Enhanced brain distribution of modified aspartoacylase.

Poddar NK, Zano S, Natarajan R, Yamamoto B, Viola RE.

Mol Genet Metab. 2014 Nov;113(3):219-24. doi: 10.1016/j.ymgme.2014.07.002. Epub 2014 Jul 12.

3.

Mutational analysis of aspartoacylase: implications for Canavan disease.

Hershfield JR, Pattabiraman N, Madhavarao CN, Namboodiri MA.

Brain Res. 2007 May 7;1148:1-14. Epub 2007 Mar 3.

4.

Characterization of human aspartoacylase: the brain enzyme responsible for Canavan disease.

Le Coq J, An HJ, Lebrilla C, Viola RE.

Biochemistry. 2006 May 9;45(18):5878-84.

5.

Structure of aspartoacylase, the brain enzyme impaired in Canavan disease.

Bitto E, Bingman CA, Wesenberg GE, McCoy JG, Phillips GN Jr.

Proc Natl Acad Sci U S A. 2007 Jan 9;104(2):456-61. Epub 2006 Dec 28.

6.

Knock-out mouse for Canavan disease: a model for gene transfer to the central nervous system.

Matalon R, Rady PL, Platt KA, Skinner HB, Quast MJ, Campbell GA, Matalon K, Ceci JD, Tyring SK, Nehls M, Surendran S, Wei J, Ezell EL, Szucs S.

J Gene Med. 2000 May-Jun;2(3):165-75.

PMID:
10894262
7.

Clinical protocol. Gene therapy of Canavan disease: AAV-2 vector for neurosurgical delivery of aspartoacylase gene (ASPA) to the human brain.

Janson C, McPhee S, Bilaniuk L, Haselgrove J, Testaiuti M, Freese A, Wang DJ, Shera D, Hurh P, Rupin J, Saslow E, Goldfarb O, Goldberg M, Larijani G, Sharrar W, Liouterman L, Camp A, Kolodny E, Samulski J, Leone P.

Hum Gene Ther. 2002 Jul 20;13(11):1391-412.

PMID:
12162821
8.

Relationship between enzyme properties and disease progression in Canavan disease.

Zano S, Wijayasinghe YS, Malik R, Smith J, Viola RE.

J Inherit Metab Dis. 2013 Jan;36(1):1-6. doi: 10.1007/s10545-012-9520-z. Epub 2012 Aug 1. Erratum in: J Inherit Metab Dis. 2013 Jan;36(1):159-60.

PMID:
22850825
9.

Adeno-associated virus-mediated aspartoacylase gene transfer to the brain of knockout mouse for canavan disease.

Matalon R, Surendran S, Rady PL, Quast MJ, Campbell GA, Matalon KM, Tyring SK, Wei J, Peden CS, Ezell EL, Muzyczka N, Mandel RJ.

Mol Ther. 2003 May;7(5 Pt 1):580-7.

10.

Canavan disease: biochemical and molecular studies.

Matalon R, Kaul R, Michals K.

J Inherit Metab Dis. 1993;16(4):744-52. Review.

PMID:
8412017
11.

Aspartoacylase gene transfer to the mammalian central nervous system with therapeutic implications for Canavan disease.

Leone P, Janson CG, Bilaniuk L, Wang Z, Sorgi F, Huang L, Matalon R, Kaul R, Zeng Z, Freese A, McPhee SW, Mee E, During MJ.

Ann Neurol. 2000 Jul;48(1):27-38. Erratum in: Ann Neurol 2000 Sep;48(3):398. Bilianuk L [corrected to Bilaniuk L].

PMID:
10894213
12.

Reexamination of aspartoacylase: is this human enzyme really a glycoprotein?

Wang Q, Viola RE.

Arch Biochem Biophys. 2014 Apr 15;548:66-73. doi: 10.1016/j.abb.2014.03.001. Epub 2014 Mar 13.

PMID:
24632142
13.

Spongy degeneration of the brain, Canavan disease: biochemical and molecular findings.

Matalon RM, Michals-Matalon K.

Front Biosci. 2000 Mar 1;5:D307-11. Review.

PMID:
10704428
14.

Aspartoacylase catalytic deficiency as the cause of Canavan disease: a structural perspective.

Wijayasinghe YS, Pavlovsky AG, Viola RE.

Biochemistry. 2014 Aug 5;53(30):4970-8. doi: 10.1021/bi500719k. Epub 2014 Jul 16.

PMID:
25003821
15.

Canavan disease: diagnosis and molecular analysis.

Matalon R.

Genet Test. 1997;1(1):21-5. Review.

PMID:
10464621
16.

Identification of the zinc binding ligands and the catalytic residue in human aspartoacylase, an enzyme involved in Canavan disease.

Herga S, Berrin JG, Perrier J, Puigserver A, Giardina T.

FEBS Lett. 2006 Oct 30;580(25):5899-904. Epub 2006 Oct 2.

17.

Aspartoacylase gene knockout in the mouse: impact on reproduction.

Surendran S, Szucs S, Tyring SK, Matalon R.

Reprod Toxicol. 2005 Jul-Aug;20(2):281-3.

PMID:
15907664
18.

Metabolic acetate therapy improves phenotype in the tremor rat model of Canavan disease.

Arun P, Madhavarao CN, Moffett JR, Hamilton K, Grunberg NE, Ariyannur PS, Gahl WA, Anikster Y, Mog S, Hallows WC, Denu JM, Namboodiri AM.

J Inherit Metab Dis. 2010 Jun;33(3):195-210. doi: 10.1007/s10545-010-9100-z. Epub 2010 May 13.

19.

Aspartoacylase is restricted primarily to myelin synthesizing cells in the CNS: therapeutic implications for Canavan disease.

Kirmani BF, Jacobowitz DM, Kallarakal AT, Namboodiri MA.

Brain Res Mol Brain Res. 2002 Nov 15;107(2):176-82.

PMID:
12487123
20.

Purification and preliminary characterization of brain aspartoacylase.

Moore RA, Le Coq J, Faehnle CR, Viola RE.

Arch Biochem Biophys. 2003 May 1;413(1):1-8.

PMID:
12706335

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