One-off liver directed AAV gene therapy achieves long term...

Molecular Genetics and Metabolism 2020 / 02 Vol. 129; Iss. 2

One-off liver directed AAV gene therapy achieves long term uptake of acid beta-glucocerebrosidase by macrophages of affected tissues in Gaucher disease

Miranda, Carlos J., Chisari, Elisa, Northcott, Natalie, Pandya, Jalpa, Liou, Benjamin, Fannin, Venette, Blackwood, Rachel, Dane, Allison, Kia, Azadeh, Canavese, Miriam, Khinder, Jaminder, Sheridan, Ro
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Volume:
129
Journal:
Molecular Genetics and Metabolism
DOI:
10.1016/j.ymgme.2019.11.283
Date:
February, 2020
File:
PDF, 332 KB
2020
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