Matches in SemOpenAlex for { <https://semopenalex.org/work/W2995083313> ?p ?o ?g. }
- W2995083313 abstract "Recombinant adeno-associated virus vectors (rAAVs), based on AAV8 and AAVrh.10, have been utilized in multiple clinical trials to treat different monogenetic diseases. The closely related AAVrh.39 has also shown promise in vivo . As recently attained for other AAV biologics, e.g., Luxturna and Zolgensma, based on AAV2 and AAV9, respectively, the vectors in this study will likely gain U.S. Food and Drug Administration approval for commercialization in the near future. This study characterized the capsid structures of these clinical vectors at atomic resolution using cryo-electron microscopy and image reconstruction for comparative analysis. The analysis suggested two key residues, S269 and N472, as determinants of BBB crossing for AAVrh.10 and AAVrh.39, a feature utilized for central nervous system delivery of therapeutic genes. The structure information thus provides a platform for engineering to improve receptor retargeting or tissue specificity. These are important challenges in the field that need attention. Capsid structure information also provides knowledge potentially applicable for regulatory product approval." @default.
- W2995083313 created "2019-12-26" @default.
- W2995083313 creator A5009470292 @default.
- W2995083313 creator A5017461125 @default.
- W2995083313 creator A5031006237 @default.
- W2995083313 creator A5032849827 @default.
- W2995083313 creator A5040613132 @default.
- W2995083313 creator A5043060351 @default.
- W2995083313 creator A5045975442 @default.
- W2995083313 creator A5054521114 @default.
- W2995083313 creator A5054738613 @default.
- W2995083313 creator A5083178801 @default.
- W2995083313 date "2020-02-28" @default.
- W2995083313 modified "2023-10-02" @default.
- W2995083313 title "Comparative Analysis of the Capsid Structures of AAVrh.10, AAVrh.39, and AAV8" @default.
- W2995083313 cites W124314106 @default.
- W2995083313 cites W1505196125 @default.
- W2995083313 cites W1507799217 @default.
- W2995083313 cites W1781789881 @default.
- W2995083313 cites W1832822495 @default.
- W2995083313 cites W1912917421 @default.
- W2995083313 cites W1965786463 @default.
- W2995083313 cites W1969538192 @default.
- W2995083313 cites W1971916603 @default.
- W2995083313 cites W1979487705 @default.
- W2995083313 cites W1980885982 @default.
- W2995083313 cites W1982698867 @default.
- W2995083313 cites W1984002179 @default.
- W2995083313 cites W1989184432 @default.
- W2995083313 cites W1990700578 @default.
- W2995083313 cites W1996745685 @default.
- W2995083313 cites W2006244553 @default.
- W2995083313 cites W2008389564 @default.
- W2995083313 cites W2009285158 @default.
- W2995083313 cites W2012799865 @default.
- W2995083313 cites W2018665497 @default.
- W2995083313 cites W2034346308 @default.
- W2995083313 cites W2038691967 @default.
- W2995083313 cites W2045600222 @default.
- W2995083313 cites W2046182783 @default.
- W2995083313 cites W2046810924 @default.
- W2995083313 cites W2048382073 @default.
- W2995083313 cites W2048651210 @default.
- W2995083313 cites W2053706844 @default.
- W2995083313 cites W2055777720 @default.
- W2995083313 cites W2057548071 @default.
- W2995083313 cites W2062871072 @default.
- W2995083313 cites W2066015597 @default.
- W2995083313 cites W2076449818 @default.
- W2995083313 cites W2079182646 @default.
- W2995083313 cites W2090600196 @default.
- W2995083313 cites W2092420017 @default.
- W2995083313 cites W2098414451 @default.
- W2995083313 cites W2100455255 @default.
- W2995083313 cites W2103918419 @default.
- W2995083313 cites W2105017686 @default.
- W2995083313 cites W2111051741 @default.
- W2995083313 cites W2113637218 @default.
- W2995083313 cites W2120683337 @default.
- W2995083313 cites W2122236774 @default.
- W2995083313 cites W2123260147 @default.
- W2995083313 cites W2123540768 @default.
- W2995083313 cites W2131971443 @default.
- W2995083313 cites W2132629607 @default.
- W2995083313 cites W2133251787 @default.
- W2995083313 cites W2134321093 @default.
- W2995083313 cites W2134766536 @default.
- W2995083313 cites W2136085939 @default.
- W2995083313 cites W2141236766 @default.
- W2995083313 cites W2141797403 @default.
- W2995083313 cites W2144081223 @default.
- W2995083313 cites W2144998676 @default.
- W2995083313 cites W2149525061 @default.
- W2995083313 cites W2149678280 @default.
- W2995083313 cites W2153140538 @default.
- W2995083313 cites W2154329712 @default.
- W2995083313 cites W2156683693 @default.
- W2995083313 cites W2157125849 @default.
- W2995083313 cites W2161655523 @default.
- W2995083313 cites W2166540858 @default.
- W2995083313 cites W2166739815 @default.
- W2995083313 cites W2170490863 @default.
- W2995083313 cites W2180229411 @default.
- W2995083313 cites W2266837556 @default.
- W2995083313 cites W2311083255 @default.
- W2995083313 cites W2520782926 @default.
- W2995083313 cites W2578185268 @default.
- W2995083313 cites W2593511418 @default.
- W2995083313 cites W2600640792 @default.
- W2995083313 cites W2767047931 @default.
- W2995083313 cites W2793421058 @default.
- W2995083313 cites W2803869264 @default.
- W2995083313 cites W2892057566 @default.
- W2995083313 cites W2897486240 @default.
- W2995083313 cites W2911059523 @default.
- W2995083313 cites W2911892982 @default.
- W2995083313 cites W2921621835 @default.
- W2995083313 cites W2939251998 @default.
- W2995083313 cites W2942547822 @default.
- W2995083313 cites W2953320394 @default.