Title: | A Neuron-Specific Gene Therapy Relieves Motor Deficits in Pompe Disease Mice |
Journal : | Molecular neurobiology, 55, 6 |
Authors: | Lee NC, Author ; Hwu WL ; Muramatsu SI ; Falk DJ ; Byrne BJ ; Cheng CH ; Shih NC ; Chang KL ; Tsai LK ; Chien YH |
Material Type: | Article |
Publication Date: | 2018 |
Size: | p 5299 |
Languages: | English |
Keywords : | acid alpha-glucosidase ; adenoassociated vectors ; central nervous system ; GAA gene ; gene therapy ; glycogen ; immunohistochemistry ; intracerebral injection ; motor skills ; mouse model ; nervous system histopathology ; neurological features ; Pompe disease ; preclinical trials |
Keywords: | Adeno-associated viral vector;Enzyme replacement therapy;Gene therapy;Glycogen;Neuron-specific;Pompe disease; |
Pubmed / DOI : | DOI : 10.1007/s12035-017-0763-4 / Pubmed : 28895054 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/28895054 |