Liver targeted gene therapy: Insights into emerging therapies-Reference-Cited by-同舟云学术

Liver targeted gene therapy: Insights into emerging therapies

Published:2019-12 Issue: Volume:34 Page:9-19
ISSN:1740-6749
Container-title:Drug Discovery Today: Technologies
language:en
Short-container-title:Drug Discovery Today: Technologies

Author:

Moscoso Carlos G.^ORCID,Steer Clifford J.

Publisher

Elsevier BV

Subject

Drug Discovery,Molecular Medicine

Reference92 articles.

1. Molecular reconstruction of Sleeping Beauty, a Tc1-like transposon from fish, and its transposition in human cells;Ivics;Cell,1997

2. The Sleeping Beauty transposon system: a non-viral vector for gene therapy;Aronovich;Hum Mol Genet,2011

3. Phenotypic correction and long-term expression of factor VIII in hemophilic mice by immunotolerization and nonviral gene transfer using the Sleeping Beauty transposon system;Ohlfest;Blood,2005

4. Nanocapsule-delivered Sleeping Beauty mediates therapeutic Factor VIII expression in liver sinusoidal endothelial cells of hemophilia A mice;Kren;J Clin Invest,2009

5. Somatic integration and long-term transgene expression in normal and haemophilic mice using a DNA transposon system;Yant;Nat Genet,2000

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