Format

Send to

Choose Destination
Methods Mol Biol. 2019;1950:333-360. doi: 10.1007/978-1-4939-9139-6_20.

AAV-Mediated Gene Delivery to the Liver: Overview of Current Technologies and Methods.

Author information

1
Department of Pediatrics, University of Florida, Gainesville, FL, USA.
2
Department of Pediatrics, Indiana University, Indianapolis, IN, USA.
3
Department of Pediatrics, Indiana University, Indianapolis, IN, USA. dmarkusi@iu.edu.

Abstract

Adeno-associated virus (AAV) vectors to treat liver-specific genetic diseases are the focus of several ongoing clinical trials. The ability to give a peripheral injection of virus that will successfully target the liver is one of many attractive features of this technology. Although initial studies of AAV liver gene transfer revealed some limitations, extensive animal modeling and further clinical development have helped solve some of these issues, resulting in several successful clinical trials that have reached curative levels of clotting factor expression in hemophilia. Looking beyond gene replacement, recent technologies offer the possibility for AAV liver gene transfer to directly repair deficient genes and potentially treat autoimmune disease.

KEYWORDS:

Adeno-associated virus; Gene delivery; Hemophilia; Hepatocyte-specific promoter; Hepatocytes; Immune tolerance; Inherited metabolic disease; Liver gene therapy

PMID:
30783984
DOI:
10.1007/978-1-4939-9139-6_20
[Indexed for MEDLINE]

Supplemental Content

Full text links

Icon for Springer
Loading ...
Support Center