Tecedor, Luis; Stein, Colleen S; Schultz, Mark L et al. (2013) CLN3 loss disturbs membrane microdomain properties and protein transport in brain endothelial cells. J Neurosci 33:18065-79
|
Benraiss, Abdellatif; Toner, Michael J; Xu, Qiwu et al. (2013) Sustained mobilization of endogenous neural progenitors delays disease progression in a transgenic model of Huntington's disease. Cell Stem Cell 12:787-99
|
Hudry, Eloise; Dashkoff, Jonathan; Roe, Alysson D et al. (2013) Gene transfer of human Apoe isoforms results in differential modulation of amyloid deposition and neurotoxicity in mouse brain. Sci Transl Med 5:212ra161
|
Chen, Yong Hong; Claflin, Kristin; Geoghegan, James C et al. (2012) Sialic acid deposition impairs the utility of AAV9, but not peptide-modified AAVs for brain gene therapy in a mouse model of lysosomal storage disease. Mol Ther 20:1393-9
|
Schultz, Mark L; Tecedor, Luis; Chang, Michael et al. (2011) Clarifying lysosomal storage diseases. Trends Neurosci 34:401-10
|
Chen, Yong Hong; Chang, Michael; Davidson, Beverly L (2009) Molecular signatures of disease brain endothelia provide new sites for CNS-directed enzyme therapy. Nat Med 15:1215-8
|
Chang, Michael; Cooper, Jonathan D; Sleat, David E et al. (2008) Intraventricular enzyme replacement improves disease phenotypes in a mouse model of late infantile neuronal ceroid lipofuscinosis. Mol Ther 16:649-56
|
Cabrera-Salazar, Mario A; Roskelley, Eric M; Bu, Jie et al. (2007) Timing of therapeutic intervention determines functional and survival outcomes in a mouse model of late infantile batten disease. Mol Ther 15:1782-8
|
Sands, Mark S; Davidson, Beverly L (2006) Gene therapy for lysosomal storage diseases. Mol Ther 13:839-49
|
Kang, Yubin; Xie, Litao; Tran, Diane Thi et al. (2005) Persistent expression of factor VIII in vivo following nonprimate lentiviral gene transfer. Blood 106:1552-8
|
Showing the most recent 10 out of 33 publications