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Items: 1 to 20 of 107

1.

Gene therapy for Canavan's disease takes a step forward.

Ahmed SS, Gao G.

Mol Ther. 2013 Mar;21(3):505-6. doi: 10.1038/mt.2013.25. No abstract available.

2.

Homozygosity for mutation G212A of the gene for aspartoacylase is associated with atypical form of Canavan's disease.

Velinov M, Zellers N, Styles J, Wisniewski K.

Clin Genet. 2008 Mar;73(3):288-9. Epub 2007 Dec 6. No abstract available.

PMID:
18070137
3.

Mild-onset presentation of Canavan's disease associated with novel G212A point mutation in aspartoacylase gene.

Janson CG, Kolodny EH, Zeng BJ, Raghavan S, Pastores G, Torres P, Assadi M, McPhee S, Goldfarb O, Saslow B, Freese A, Wang DJ, Bilaniuk L, Shera D, Leone P.

Ann Neurol. 2006 Feb;59(2):428-31.

PMID:
16437572
4.

Dietary treatment proposed for Canavan's disease.

Chen V.

Lancet Neurol. 2005 May;4(5):273. No abstract available.

PMID:
15861552
5.

Global CNS gene transfer for a childhood neurogenetic enzyme deficiency: Canavan disease.

Leone P, Janson CG, McPhee SJ, During MJ.

Curr Opin Mol Ther. 1999 Aug;1(4):487-92. Review.

PMID:
11713764
6.

Molecular basis of Canavan's disease: from human to mouse.

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

J Child Neurol. 2003 Sep;18(9):604-10. Review.

PMID:
14572138
7.

A single intravenous rAAV injection as late as P20 achieves efficacious and sustained CNS Gene therapy in Canavan mice.

Ahmed SS, Li H, Cao C, Sikoglu EM, Denninger AR, Su Q, Eaton S, Liso Navarro AA, Xie J, Szucs S, Zhang H, Moore C, Kirschner DA, Seyfried TN, Flotte TR, Matalon R, Gao G.

Mol Ther. 2013 Dec;21(12):2136-47. doi: 10.1038/mt.2013.138. Epub 2013 Jul 2.

8.

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.

PMID:
12718900
9.

Recent advances in Canavan disease.

Matalon R, Michals-Matalon K.

Adv Pediatr. 1999;46:493-506. Review.

PMID:
10645473
10.

Current development of adeno-associated viral vectors.

Romano G.

Drug News Perspect. 2005 Jun;18(5):311-6. Review.

PMID:
16193103
11.

Case 15-1998: elevated N-acetylaspartic acid activity in Canavan's disease.

Breslau J.

N Engl J Med. 1998 Aug 27;339(9):636. No abstract available.

12.
13.

Novel mutation of aspartoacylase gene in a Turkish patient with Canavan disease.

Unalp A, Altiok E, Uran N, Oztürk A, Yüksel S.

J Trop Pediatr. 2008 Jun;54(3):208-10. Epub 2007 Nov 12.

PMID:
17999961
14.

The impact of structural biology on neurobiology.

Viola RE.

Proc Natl Acad Sci U S A. 2007 Jan 9;104(2):399-400. Epub 2007 Jan 3. No abstract available.

15.

Molecular basis of Canavan disease.

Matalon R, Michals-Matalon K.

Eur J Paediatr Neurol. 1998;2(2):69-76. Review. No abstract available.

PMID:
10724099
16.

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
17.

Metabolic changes in the knockout mouse for Canavan's disease: implications for patients with Canavan's disease.

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

J Child Neurol. 2003 Sep;18(9):611-5.

PMID:
14572139
18.

Restoration of aspartoacylase activity in CNS neurons does not ameliorate motor deficits and demyelination in a model of Canavan disease.

Klugmann M, Leichtlein CB, Symes CW, Serikawa T, Young D, During MJ.

Mol Ther. 2005 May;11(5):745-53.

PMID:
15851013
19.

Defective N-acetylaspartate catabolism reduces brain acetate levels and myelin lipid synthesis in Canavan's disease.

Madhavarao CN, Arun P, Moffett JR, Szucs S, Surendran S, Matalon R, Garbern J, Hristova D, Johnson A, Jiang W, Namboodiri MA.

Proc Natl Acad Sci U S A. 2005 Apr 5;102(14):5221-6. Epub 2005 Mar 22.

20.

Prenatal diagnosis of Canavan disease--problems and dilemmas.

Besley GT, Elpeleg ON, Shaag A, Manning NJ, Jakobs C, Walter JH.

J Inherit Metab Dis. 1999 May;22(3):263-6. No abstract available.

PMID:
10384383
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