scholarly journals Similar Therapeutic Efficacy Between a Single Administration of Gene Therapy and Multiple Administrations of Recombinant Enzyme in a Mouse Model of Lysosomal Storage Disease

2014 ◽  
Vol 25 (7) ◽  
pp. 609-618 ◽  
Author(s):  
Rita Ferla ◽  
Pamela Claudiani ◽  
Gabriella Cotugno ◽  
Paola Saccone ◽  
Elvira De Leonibus ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Mouse Model ◽  
Lysosomal Storage Disease ◽  
Therapeutic Efficacy ◽  
Storage Disease ◽  
Recombinant Enzyme ◽  
Single Administration ◽  
Lysosomal Storage

scholarly journals Low-dose Gene Therapy Reduces the Frequency of Enzyme Replacement Therapy in a Mouse Model of Lysosomal Storage Disease

2016 ◽  
Vol 24 (12) ◽  
pp. 2054-2063 ◽  
Author(s):  
Marialuisa Alliegro ◽  
Rita Ferla ◽  
Edoardo Nusco ◽  
Chiara De Leonibus ◽  
Carmine Settembre ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Mouse Model ◽  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Lysosomal Storage Disease ◽  
Low Dose ◽  
Storage Disease ◽  
Lysosomal Storage ◽  
Enzyme Replacement

scholarly journals New Players in the Infertility of a Mouse Model of Lysosomal Storage Disease: The Hypothalamus-Pituitary-Gonadal Axis

2014 ◽  
Vol 4 ◽  
Author(s):  
Paola Piomboni ◽  
Laura Governini ◽  
Martina Gori ◽  
Erica Puggioni ◽  
Elvira Costantino-Ceccarini ◽  
...  
Keyword(s):  
Mouse Model ◽  
Lysosomal Storage Disease ◽  
Storage Disease ◽  
Lysosomal Storage

scholarly journals Gene Therapy Approaches for Lysosomal Storage Disease: Next-Generation Treatment

2012 ◽  
Vol 23 (8) ◽  
pp. 808-815 ◽  
Author(s):  
Barry J. Byrne ◽  
Darin J. Falk ◽  
Nathalie Clément ◽  
Cathryn S. Mah
Keyword(s):  
Gene Therapy ◽  
Lysosomal Storage Disease ◽  
Storage Disease ◽  
Next Generation ◽  
Lysosomal Storage
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