710. Optimization of AAV Vector Design for Safe Expression...

Molecular Therapy 2015 / 05 Vol. 23

710. Optimization of AAV Vector Design for Safe Expression of β-N-Acetylhexosaminidase in the Brain for Tay-Sachs Disease Gene Therapy

Golebiowski, Diane, Petrosky, Keiko, Van der Marel, Kajo, Hutto, Elizabeth, Curran, Elizabeth, Balkanska-Sinclair, Elena, Bishop, Nina, Santos, Rosemary, Cummings Macri, Sheila, Martin, Douglas, Gouni
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Volume:
23
Language:
english
Journal:
Molecular Therapy
DOI:
10.1016/s1525-0016(16)34319-2
Date:
May, 2015
File:
PDF, 278 KB
english, 2015
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