scholarly journals 210. AAV Vector-Mediated Enzyme Replacement Therapy Is Efficacious in Reversing Storage Pathology and Preventing Purkinje Cell Death in a Mouse Model of Niemann-Pick A Disease

2004 ◽  
Vol 9 ◽  
pp. S81
Keyword(s):  
Cell Death ◽  
Mouse Model ◽  
Enzyme Replacement Therapy ◽  
Purkinje Cell ◽  
Replacement Therapy ◽  
Aav Vector ◽  
Enzyme Replacement ◽  
Purkinje Cell Death ◽  
Niemann Pick

Enzyme replacement therapy in a mouse model of aspartylglycosaminuria

2000 ◽  
Vol 14 (2) ◽  
pp. 361-367 ◽  
Author(s):  
Ulla Dunder ◽  
Vesa Kaartinen ◽  
Pirjo Valtonen ◽  
Eira VÄÄnÄnen ◽  
Veli-Matti Kosma ◽  
...  
Keyword(s):  
Mouse Model ◽  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Enzyme Replacement

scholarly journals Galactosialidosis: preclinical enzyme replacement therapy in a mouse model of the disease, a proof of concept

2021 ◽  
Vol 20 ◽  
pp. 191-203
Author(s):  
Jaclyn Cadaoas ◽  
Huimin Hu ◽  
Gabrielle Boyle ◽  
Elida Gomero ◽  
Rosario Mosca ◽  
...  
Keyword(s):  
Mouse Model ◽  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Proof Of Concept ◽  
Enzyme Replacement

scholarly journals Correction of CNS defects in the MPSII mouse model via systemic enzyme replacement therapy

2010 ◽  
Vol 19 (24) ◽  
pp. 4871-4885 ◽  
Author(s):  
Vinicia Assunta Polito ◽  
Serena Abbondante ◽  
Roman S. Polishchuk ◽  
Edoardo Nusco ◽  
Rosaria Salvia ◽  
...  
Keyword(s):  
Mouse Model ◽  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Enzyme Replacement

scholarly journals Enzyme replacement therapy on hypophosphatasia mouse model

2013 ◽  
Vol 37 (2) ◽  
pp. 309-317 ◽  
Author(s):  
Hirotaka Oikawa ◽  
Shunji Tomatsu ◽  
Bisong Haupt ◽  
Adriana M. Montaño ◽  
Tsutomu Shimada ◽  
...  
Keyword(s):  
Mouse Model ◽  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Enzyme Replacement

scholarly journals Intracisternal cyclodextrin prevents cerebellar dysfunction and Purkinje cell death in feline Niemann-Pick type C1 disease

2015 ◽  
Vol 7 (276) ◽  
pp. 276ra26-276ra26 ◽  
Author(s):  
Charles H. Vite ◽  
Jessica H. Bagel ◽  
Gary P. Swain ◽  
Maria Prociuk ◽  
Tracey U. Sikora ◽  
...  
Keyword(s):  
Cell Death ◽  
Purkinje Cell ◽  
Cerebellar Dysfunction ◽  
Purkinje Cell Death ◽  
Niemann Pick

Reduced glucosylceramide in the mouse model of Fabry disease: Correction by successful enzyme replacement therapy

Gene ◽  
2014 ◽  
Vol 536 (1) ◽  
pp. 97-104 ◽  
Author(s):  
Rui Quinta ◽  
Daniel Rodrigues ◽  
Marisa Assunção ◽  
Maria Fatima Macedo ◽  
Olga Azevedo ◽  
...  
Keyword(s):  
Mouse Model ◽  
Enzyme Replacement Therapy ◽  
Fabry Disease ◽  
Replacement Therapy ◽  
Enzyme Replacement
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