SC-AAV腺相关病毒载体质粒 BioVector NTCC质粒载体菌种细胞基因保藏中心

SC-AAV腺相关病毒载体质粒

Self-complementary adeno-associated virus (scAAV) is a viral vector engineered from the naturally occurring adeno-associated virus (AAV) to be used as a tool for gene therapy.[1] Use of recombinant AAV (rAAV) has been successful in clinical trials addressing a variety of diseases.
AAV Helper-Free System allows the production of infectious recombinant human adenoassociated virus (rAAV) virions without the use of a helper virus (Figure 3). In the AAV Helper-Free
System, most of the adenovirus gene products required for the production of infective AAV particles
are supplied on the plasmid pHelper (i.e. E2A, E4, and VA RNA genes) that is co-transfected into cells
with human AAV vector DNA. The remaining adenoviral gene product is supplied by the 293 host
cells, which stably express the adenovirus E1 gene. By eliminating the requirement for live helper
virus the AAV Helper-Free System provides a safer and more convenient gene delivery system. In the
AAV Helper-Free System, the rep and cap genes have been removed from the viral vector that
contains AAV-2 ITRs and are supplied in trans on the plasmid pAAV-RC. The removal of the AAV
rep and cap genes allows for insertion of a gene of interest in the viral genome.

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