Correction of pathology in mice displaying Gaucher disease type 1 by a clinically-applicable lentiviral vector.

Dahl, Maria; Smith, Emma M K; Warsi, Sarah; Rothe, Michael; Ferraz, Maria J; Aerts, Johannes M F G; Golipour, Azadeh; Harper, Claudia; Pfeifer, Richard; Pizzurro, Daniella; Schambach, Axel; Mason, Chris; Karlsson, Stefan.

Afiliação

Dahl M; Department of Molecular Medicine and Gene Therapy, Lund University, Lund, Sweden.
Smith EMK; Department of Molecular Medicine and Gene Therapy, Lund University, Lund, Sweden.
Warsi S; Department of Molecular Medicine and Gene Therapy, Lund University, Lund, Sweden.
Rothe M; Hannover Medical School, Institute of Experimental Hematology, Hannover, Germany.
Ferraz MJ; Department of Medical Biochemistry, Leiden University, Leiden, the Netherlands.
Aerts JMFG; Department of Medical Biochemistry, Leiden University, Leiden, the Netherlands.
Golipour A; AVROBIO, Inc., Cambridge, MA, USA.
Harper C; AVROBIO, Inc., Cambridge, MA, USA.
Pfeifer R; AVROBIO, Inc., Cambridge, MA, USA.
Pizzurro D; AVROBIO, Inc., Cambridge, MA, USA.
Schambach A; Hannover Medical School, Institute of Experimental Hematology, Hannover, Germany.
Mason C; Division of Hematology/Oncology, Boston's Children's Hospital, Harvard Medical School, Boston, MA, USA.
Karlsson S; AVROBIO, Inc., Cambridge, MA, USA.

Mol Ther Methods Clin Dev ; 20: 312-323, 2021 Mar 12.

Article em En | MEDLINE | ID: mdl-33511245

Texto completo: 1 Base de dados: MEDLINE Idioma: En Ano de publicação: 2021 Tipo de documento: Article

Texto completo: 1 Base de dados: MEDLINE Idioma: En Ano de publicação: 2021 Tipo de documento: Article