Gene Transfer in the Liver Using Recombinant Adeno-Associated Virus

Gene Transfer in the Liver Using Recombinant Adeno-Associated Virus

Gene Transfer in the Liver Using Recombinant Adeno-Associated Virus

ABSTRACT

Liver-directed gene transfer and gene therapy are rapidly gaining attention primarily
because the liver is centrally involved in a variety of metabolic functions that are affected
in various inherited disorders. Recombinant adeno-associated virus (rAAV) is a popular
gene delivery vehicle for gene therapy, and intravenous delivery of some rAAV serotypes
results in very efficient transduction in the liver. rAAV-mediated gene transfer to the liver
can be used to create somatic transgenic animals or disease models for studying the function
of various genes and miRNAs. The liver is the target tissue for gene therapy of many
inborn metabolic diseases and may also be exploited as a “biofactory” for production of
coagulation factors, insulin, growth hormones, and other non-hepatic proteins. Hence,
efficient delivery of transgenes and small RNAs to the liver by rAAV vectors has been
of long-standing interest to research scientists and clinicians alike. This unit describes
methods for delivery of rAAV vectors by several injection routes, followed by a range
of analytical methods for assessing the expression, activity, and effects of the transgene
and its product.
read online

Post Comment