Biomarkers for disease progression and AAV therapeutic efficacy in feline Sandhoff disease

Por Bradbury, Allison M, Gray-Edwards, Heather L, Shirley, Jamie L, McCurdy, Victoria J, Colaco, Alexandria N, Randle, Ashley N, Christopherson, Pete W, Bird, Allison C, Johnson, Aime K, Wilson, Diane U, Hudson, Judith A, De Pompa, Nicholas L, Sorjonen, Donald C, Brunson, Brandon L, Jeyakumar, Mylvaganam, Platt, Frances M, Baker, Henry J, Cox, Nancy R, Sena-Esteves, Miguel, Martin, Douglas R
Publicado no Exp Neurol (2014)

Novel Biomarkers of Human GM1 Gangliosidosis Reflect the Clinical Efficacy of Gene Therapy in a Feline Model

Por Gray-Edwards, Heather L., Regier, Debra S., Shirley, Jamie L., Randle, Ashley N., Salibi, Nouha, Thomas, Sarah E., Latour, Yvonne L., Johnston, Jean, Golas, Gretchen, Maguire, Annie S., Taylor, Amanda R., Sorjonen, Donald C., McCurdy, Victoria J., Christopherson, Peter W., Bradbury, Allison M., Beyers, Ronald J., Johnson, Aime K., Brunson, Brandon L., Cox, Nancy R., Baker, Henry J., Denney, Thomas S., Sena-Esteves, Miguel, Tifft, Cynthia J., Martin, Douglas R.
Publicado no Mol Ther (2017)