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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.
This lab-made progeny of rAAV is termed "self-complementary" because the coding region has been designed to form an intra-molecular double-stranded DNA template. A rate-limiting step for the standard AAV genome involves the second-strand synthesis since the typical AAV genome is a single-stranded DNA template.
It was found that AAV genome inserts in less than ~10% of occasions AAV infects a cell and the expression is less than when episomally expressed. [4] AAV can only package genomes between 2 – 5.2 kb in size when they are flanked with inverted terminal repeat sequences (ITRs), but optimally holds a genome of 4.1 to 4.9 kb in length. [ 5 ]
Alipogene tiparvovec (Glybera): AAV-based treatment for lipoprotein lipase deficiency (no longer commercially available) Axicabtagene ciloleucel (Yescarta): treatment for large B-cell lymphoma [1] Beremagene geperpavec (Vyjuvek): treatment of wounds. [2] Betibeglogene autotemcel (Zynteglo): treatment for beta thalassemia [3]
A larger number of mRNAs would express a greater amount of protein, and how many copies of mRNA are generated depends on the promoter used in the vector. [12] The expression may be constitutive, meaning that the protein is produced constantly in the background, or it may be inducible whereby the protein is expressed only under certain condition ...
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