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Phase 2–3 gene therapy trial using adeno-associated virus vector for patients with mucopolysaccharidosis type IIIA
Phase 2–3 gene therapy trial using adeno-associated virus vector for patients with mucopolysaccharidosis type IIIA
2020
Frits A. Wijburg
Kristian Aquilina
Bénédicte Héron
Anupam Chakrapani
Ron Crystal
Timothee de Saint Denis
Lisa T. Emrick
Daniel J. Guillaume
Nicole Muschol
Marc Souweidane
Raymond Y. Wang
Michel Zerah
Chester B. Whitley
Sophie Olivier
Keywords:
Virology
Genetics
Diabetes mellitus
Mucopolysaccharidosis Type IIIA
Genetic enhancement
Biology
Adeno-associated virus
Correction
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