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Wild-type AAV has attracted considerable interest from gene therapy researchers due to a number of features. Chief amongst these was the virus's apparent lack of pathogenicity. It can also infect non-dividing cells and has the ability to stably integrate into the host cell genome at a specific site (designated AAVS1) in the human chromosome 19.
Onasemnogene abeparvovec is a biologic drug consisting of AAV9 virus capsids that contains a SMN1 transgene along with synthetic promoters. [5] Upon administration, the AAV9 viral vector delivers the SMN1 transgene to the affected motor neurons, where it leads to an increase in SMN protein.
Different scAAV and AAV serotypes can efficiently transfect a variety of cellular targets. [10] [11] Like all vector-based approaches to gene therapy, one obstacle in translating therapies from pre-clinical trials into a human clinical application will be the production of large quantities of highly concentrated virus. [12]
Adeno Associated Virus (AAV) (lower panel) – To produce AAV, package a gene of interest into the AAV-ITR vector and transfect cells with a Helper vector and the Rep/Cap DNA integration vector. Adeno-associated viruses (AAVs) are relatively small single-stranded DNA viruses belonging to Parvoviridae and, like lentiviral vectors, AAVs can ...
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Alipogene tiparvovec was expected to cost around US$1.6 million per treatment in 2012, [9] —revised to $1 million in 2015, [10] —making it the most expensive medicine in the world at the time. [11] However, replacement therapy, a similar treatment, can cost over $300,000 per year, for life. [4]
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