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

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AAV9 intracerebroventricular gene therapy improves lifespan, locomotor function and pathology in a mouse model of Niemann-Pick type C1 disease by Hughes, Michael P, Smith, Dave A, Morris, Lauren, Fletcher, Claire, Colaco, Alexandria, Huebecker, Mylene, Tordo, Julie, Palomar, Nuria, Massaro, Giulia, Henckaerts, Els, Waddington, Simon N, Platt, Frances M, Rahim, Ahad A

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Defective platelet function in Niemann‐Pick disease type C1 by Chen, Oscar C. W., Colaco, Alexandria, Davis, Lianne C., Kiskin, Fedir N., Farhat, Nicole Y., Speak, Anneliese O., Smith, David A., Morris, Lauren, Eden, Emily, Tynan, Patricia, Churchill, Grant C., Galione, Antony, Porter, Forbes D., Platt, Frances M.

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