Molecular Therapy - Methods & Clinical Development
 2024
DOI: 10.1016/j.omtm.2024.101200
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Biodistribution and safety of a single rAAV3B-AAT vector for silencing and replacement of alpha-1 antitrypsin in Cynomolgus macaques

Meghan Blackwood,
Alisha M. Gruntman,
Qiushi Tang
et al.
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