Title: | Translational Fidelity of Intrathecal Delivery of scAAV9-SMN1 for Spinal Muscular Atrophy. |
Journal : | Human gene therapy, 25, 7 |
Authors: | Passini MA ; Bu J ; Richards AR ; Treleaven CM ; Sullivan JA ; O'Riordan CR ; Scaria A ; Kells AP ; Samaranch L ; San Sebastian W ; Federici T ; Fiandaca MS ; Boulis NM ; Bankiewicz K ; Shihabuddin LS ; Cheng SH |
Material Type: | Article |
Publication Date: | 2014 |
Size: | p. 619-630 |
Languages: | English |
Keywords : | adenoassociated vectors ; cross-sectional study ; gene expression ; gene therapy ; motor neurons ; SMN protein ; spinal cord ; spinal muscular atrophy ; spinal muscular atrophy mouse ; therapeutic prospects |
Pubmed / DOI : | DOI : 10.1089/hum.2014.011 / Pubmed : 24617515 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/24617515 |