Lipid nanoparticle-encapsulated mRNA therapy corrects serum total bilirubin level in Crigler-Najjar syndrome mouse model.

Greig, Jenny A; Chorazeczewski, Joanna K; Chowdhary, Vivek; Smith, Melanie K; Jennis, Matthew; Tarrant, James C; Buza, Elizabeth L; Coughlan, Kimberly; Martini, Paolo G V; Wilson, James M.

Afiliação

Greig JA; Gene Therapy Program, Department of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
Chorazeczewski JK; Gene Therapy Program, Department of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
Chowdhary V; Gene Therapy Program, Department of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
Smith MK; Gene Therapy Program, Department of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
Jennis M; Gene Therapy Program, Department of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
Tarrant JC; Gene Therapy Program, Department of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
Buza EL; Gene Therapy Program, Department of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
Coughlan K; Moderna, Inc., Cambridge, MA, USA.
Martini PGV; Moderna, Inc., Cambridge, MA, USA.
Wilson JM; Gene Therapy Program, Department of Medicine, University of Pennsylvania, Philadelphia, PA, USA.