AI Article Synopsis
- Researchers developed improved AAV vectors by modifying specific surface amino acids on AAV2 and AAV3 capsids to enhance transduction efficiency using lower doses.
- They also modified the inactive AAV genome to improve transgene expression, leading to a more effective delivery system.
- The combination of these capsid and genome modifications resulted in optimized AAV vectors that can deliver genes more efficiently at reduced doses, potentially benefiting human gene therapy.
Article Abstract
We have described the development of capsid-modified next-generation AAV vectors for both AAV2 and AAV3 serotypes, in which specific surface-exposed tyrosine (Y), serine (S), threonine (T), and lysine (K) residues on viral capsids were modified to achieve high-efficiency transduction at lower doses. We have also described the development of genome-modified AAV vectors, in which the transcriptionally inactive, single-stranded AAV genome was modified to achieve improved transgene expression. Here, we describe that combination of capsid modifications and genome modifications leads to the generation of optimized AAV serotype vectors, which transduce cells and tissues more efficiently, both in vitro and in vivo, at ∼20-30-fold reduced doses. These studies have significant implications in the potential use of the optimized AAV serotype vectors in human gene therapy.
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| http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4991608 | PMC |
| http://dx.doi.org/10.1089/hgtb.2016.054 | DOI Listing |
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