Biodistribution and safety of a single rAAV3B-AAT vector for silencing and replacement of alpha-1 antitrypsin in Cynomolgus macaques.
Blackwood M, Gruntman AM, Tang Q, Pires-Ferreira D, Reil D, Kondratov O, Marsic D, Zolotukhin S, Gernoux G, Keeler AM, Mueller C, Flotte TR. Biodistribution and safety of a single rAAV3B-AAT vector for silencing and replacement of alpha-1 antitrypsin in Cynomolgus macaques. Mol Ther Methods Clin Dev. 2024 Mar 14; 32(1):101200.