Biodistribution and safety of a single rAAV3B-AAT vector for silencing and replacement of alpha-1 antitrypsin in Cynomolgus macaques

Alpha-1 antitrypsin deficiency (AATD) is characterized by both chronic lung disease due to loss of wild-type AAT (M-AAT) antiprotease function and liver disease due to toxicity from delayed secretion, polymerization, and aggregation of misfolded mutant AAT (Z-AAT). The ideal gene therapy for AATD sh...

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Veröffentlicht in:	Molecular therapy. Methods & clinical development 2024-03, Vol.32 (1), p.101200-101200, Article 101200
Hauptverfasser:	Blackwood, Meghan, Gruntman, Alisha M., Tang, Qiushi, Pires-Ferreira, Debora, Reil, Darcy, Kondratov, Oleksandr, Marsic, Damien, Zolotukhin, Sergei, Gernoux, Gwladys, Keeler, Allison M., Mueller, Christian, Flotte, Terence R.
Format:	Artikel
Sprache:	eng
Schlagworte:	AAV gene therapy AAV3B alpha-1 antitrypsin deficiency biodistribution gene silencing Human health and pathology Life Sciences miRNA Original preclinical
Online-Zugang:	Volltext
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