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Items: 5

1.

Long-term follow-up after gene therapy for canavan disease.

Leone P, Shera D, McPhee SW, Francis JS, Kolodny EH, Bilaniuk LT, Wang DJ, Assadi M, Goldfarb O, Goldman HW, Freese A, Young D, During MJ, Samulski RJ, Janson CG.

Sci Transl Med. 2012 Dec 19;4(165):165ra163. doi: 10.1126/scitranslmed.3003454.

2.

Lithium citrate reduces excessive intra-cerebral N-acetyl aspartate in Canavan disease.

Assadi M, Janson C, Wang DJ, Goldfarb O, Suri N, Bilaniuk L, Leone P.

Eur J Paediatr Neurol. 2010 Jul;14(4):354-9. doi: 10.1016/j.ejpn.2009.11.006. Epub 2010 Jan 19.

PMID:
20034825
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.

Acute pancreatitis in children from Valproic acid: case series and review.

Grauso-Eby NL, Goldfarb O, Feldman-Winter LB, McAbee GN.

Pediatr Neurol. 2003 Feb;28(2):145-8. Review.

PMID:
12699868
5.

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

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