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Pronounced Therapeutic Benefit of a Single Bidirectional AAV Vector Administered Systemically in Sandhoff Mice
Lahey, Hannah G., Webber, Chelsea J., Golebiowski, Diane, Izzo, Cassandra M., Horn, Erin, Taghian, Toloo, Rodriguez, Paola, Batista, Ana Rita, Ellis, Lauren E., Hwang, Misako, Martin, Douglas R., GrayJournal:
Molecular Therapy
DOI:
10.1016/j.ymthe.2020.06.021
Date:
June, 2020
File:
PDF, 2.11 MB
2020