Phase 2–3 gene therapy trial using adeno-associated virus...

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

Phase 2–3 gene therapy trial using adeno-associated virus vector for patients with mucopolysaccharidosis type IIIA

Wijburg, Frits, Aquilina, Kristian, Heron, Bénédicte, Chakrapani, Anupam, Crystal, Ron, de Saint Denis, Timothee, Emrick, Lisa, Guillaume, Daniel, Muschol, Nicole, Souweidane, Marc, Wang, Raymond, Z
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Volume:
129
Journal:
Molecular Genetics and Metabolism
DOI:
10.1016/j.ymgme.2019.11.432
Date:
February, 2020
File:
PDF, 331 KB
2020
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