AI Article Synopsis
- AAVs are effective and safe tools for gene delivery, with AAV2 being the most studied serotype.
- The study focused on engineering the less-explored VR-IV region of AAV2 by targeting specific amino acid positions and creating a diverse viral vector library.
- Two new variants, AAV2.A1 and AAV2.A2, showed significantly improved efficiency in transducing the central nervous system, offering promising options for brain gene therapy.
Article Abstract
Adeno-associated viruses (AAVs) have become safe and effective tools for therapeutic in vivo gene drug delivery. Among many AAV serotypes, AAV2 is the most well-characterized. Although many studies have been carried out on the engineering of the capsid VR-VIII region, few attempts have been made in the VR-IV region. Here, we targeted amino acid positions 442-469 of the VR-IV region and established an engineering paradigm of computer-aided directed evolution, based on training samples from previous datasets, to obtain a viral vector library with high diversity (~95,089). We further examined two variants selected from the library. The transduction efficiency of these two novel AAV variants, AAV2.A1 and AAV2.A2, in the central nervous system was 10-15 times higher than that of AAV2. This finding provides new vehicles for delivering gene drugs to the brain.
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Source |
|---|---|
| http://www.ncbi.nlm.nih.gov/pmc/articles/PMC10143561 | PMC |
| http://dx.doi.org/10.3390/v15040848 | DOI Listing |
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