Results 111 to 120 of about 2,664 (184)

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

, 2000
With the ultimate goal of developing safe and effective in vivo gene therapy for the treatment of Canavan disease and other neurological disorders, we developed a non-viral lipid-entrapped, polycation-condensed delivery system (LPD) for central nervous ...
Freese, A, Wang, Z, Sorgi, F, Zeng, Z, Mee, E, Bilaniuk, L, Kaul, R, Matalon, R, McPhee, S W, During, M J, Janson, C G, Leone, P, Huang, L   +12 more
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Phenotypic characterisation of the tremor mutant and AAV mediated aspartoacylase gene transfer in the rat model of Canavan disease

, 2004
Subscription resource available via Digital Dissertations only.The doctoral studies described in this thesis involve the phenotypic characterization of the tremor rat, an animal model of Canavan disease, and a proof of principle gene transfer study in ...
McPhee, Scott William John
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Human iPSC-derived neural progenitor cells rescue motor function and brain pathology in symptomatic Canavan disease mice. [PDF]

Stem Cell Reports
Jackson N, Feng L, Chao J, Ye P, Luong Q, Galo K, Sun G, Hu W, Shi Y.   +8 more
europepmc   +1 more source

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

, 1999
The neurogenetic prototypic disease on which we chose to test our gene therapy strategy is Canavan disease (CD). CD is an autosomal recessive leukodystrophy associated with spongiform degeneration of the brain.
Leone, Paola, McPhee, Scott J, During, Matthew J, Janson, Christopher G   +3 more
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Deep Intronic SVA_E Insertion Identified as the Most Common Pathogenic Variant Associated With Canavan Disease: A Diagnostic Blind Spot. [PDF]

Neurol Genet
Dominguez Gonzalez CA, Bell KM, Rajagopalan R, de Silva MG, Lemes A, Zabala C, Pérez-Vidarte F, Cerisola A, Vossough A, Whitehead MT, Cunningham C, Brown NJ, Quin R, Simons C, Conway T, Uebergang E, Rius R, Kumaheri MA, Kotes ER, Vohra A, Zalusky MPG, Anderson ZB, Storz SHR, Ward SA, Goffena J, Gustafson JA, White SM, Vanderver A, Miller DE.   +28 more
europepmc   +1 more source

Canavan disease

, 2008
Frank Gaillard, Arlene Campos, Henry Knipe   +2 more
openaire   +2 more sources

Deep Intronic SVA_E Retrotransposition as a Novel Factor in Canavan Disease Pathogenesis. [PDF]

Hum Gene Ther
Weiß M, Selig M, Friedrich J, Wierczeiko A, Diederich S, Sigel H, Bredow J, Eichler FS, Nagy A, Seyler D, Holthöfer L, Gerber S, Schweiger S, Linke M, Bley A.   +14 more
europepmc   +1 more source

Homozygous Paternally Inherited ASPA Variant in a Patient with Canavan Disease. [PDF]

Mol Syndromol
Yalcintepe S, Maras T, Kizilyar I, Sezginer Guler H, Zhuri D, Atli E, Ozen Y, Gurkan H.   +7 more
europepmc   +1 more source

Canavan Disease

, 2012
Andrea Rossi, Chen Hoffmann
openaire   +2 more sources

Myelin and re-myelination in Multiple Sclerosis and Canavan Disease

, 2014
Myelin sheath formation around axons is fundamentally important for central nervous system (CNS) function. Understanding the process of myelination, and the mechanism of immune-induced demyelination is essential in designing new therapeutic strategies ...
Mills Ko, Emily
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