Portal vein delivery of viral vectors for gene therapy for hemophilia.
Sherman, Alexandra; Schlachterman, Alexander; Cooper, Mario; Merricks, Elizabeth P; Raymer, Robin A; Bellinger, Dwight A; Herzog, Roland W; Nichols, Timothy C.
Methods Mol Biol
; 1114: 413-26, 2014.
Artigo em Inglês | MEDLINE | ID: mdl-24557919
Update on clinical gene therapy for hemophilia.
Genetic Targeting of the Albumin Locus to Treat Hemophilia.
Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant.
Long-term safety and efficacy of factor IX gene therapy in hemophilia B.
Adeno-associated viral vectors for the treatment of hemophilia.
Computationally designed liver-specific transcriptional modules and hyperactive factor IX improve hepatic gene therapy.
Eliminating HIV-1 Packaging Sequences from Lentiviral Vector Proviruses Enhances Safety and Expedites Gene Transfer for Gene Therapy.
Gene therapy for hemophilia: advancing beyond the first clinical success.
In Utero Transfer of Adeno-Associated Viral Vectors Produces Long-Term Factor IX Levels in a Cynomolgus Macaque Model.
Gene Therapy for Hemophilia.