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

The GM2 gangliosidoses, Tay–Sachs disease (TSD) and Sandhoff disease (SD), are progressive neurodegenerative disorders that are caused by a mutation in the enzyme β-N-acetylhexosaminidase (Hex). Due to the recent emergence of novel experimental treatments, biomarker development has become particular...

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Veröffentlicht in:	Experimental neurology 2015-01, Vol.263, p.102-112
Hauptverfasser:	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.
Format:	Artikel
Sprache:	eng
Schlagworte:	Adeno-associated virus Animals beta-N-Acetylhexosaminidases - administration & dosage beta-N-Acetylhexosaminidases - genetics Biomarkers Biomarkers - analysis Brain - pathology Cats Dependovirus Disease Models, Animal Disease Progression Gene therapy Genetic Therapy - methods Genetic Vectors GM2 ganglioside Hexosaminidase Leukocytes, Mononuclear - pathology Lysosomal storage disorder Lysosomes - pathology Magnetic Resonance Imaging Neurodegenerative disease Sandhoff Disease - blood Sandhoff Disease - cerebrospinal fluid Sandhoff Disease - pathology
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