The adeno-associated virus serotype 1 (AAV1) viral vector delivers an intact copy of the human lipoprotein lipase (LPL) gene to muscle cells. The LPL gene is not inserted into the cell's chromosomes but remains as free floating DNA in the nucleus. The injection is followed by immunosuppressive therapy to prevent immune reactions to the virus. Data from the clinical trials indicates that fat concentrations in blood were reduced between 3 and 12 weeks after injection, in nearly all patients. The advantages of AAV include apparent lack of pathogenicity, delivery to non-dividing cells, and much smaller risk of insertion compared to retroviruses, which show random insertion with accompanying risk of cancer. AAV also presents very low immunogenicity, mainly restricted to generating neutralising antibodies, and little well defined cytotoxic response. The cloning capacity of the vector is limited to replacement of the virus's 4.8 kilobase genome.
Alipogene tiparvovec
Valoctocogene roxaparvovec
Neo-Viagra-Y.R.
Odalasvir
Enadenotucirev
Fosamprenavir
Onasemnogene abeparvovec
Elbasvir