Targeting Root Cause by Systemic scAAV9-hIDS Gene Delivery: Functional Correction and Reversal of Severe MPS II in Mice
| Autor: | Fu, Haiyan, Zaraspe, Kim, Murakami, Naoko, Meadows, Aaron S., Pineda, Ricardo J., McCarty, Douglas M., Muenzer, Joseph |
|---|---|
| Zdroj: | In Molecular Therapy - Methods & Clinical Development 21 September 2018 10:327-340 |
| Databáze: | ScienceDirect |
| Externí odkaz: |
|