An AAV capsid reprogrammed to bind human transferrin receptor mediates brain-wide gene delivery.
Huang Q, Chan KY, Wu J, Botticello-Romero NR, Zheng Q, Lou S, Keyes C, Svanbergsson A, Johnston J, Mills A, Lin CY, Brauer PP, Clouse G, Pacouret S, Harvey JW, Beddow T, Hurley JK, Tobey IG, Powell M, Chen AT, Barry AJ, Eid FE, Chan YA, Deverman BE
Science. 2024 Jun 14;384(6701):1220-1227. doi: 10.1126/science.adm8386. Epub 2024 May 16.
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PubMed
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Article
Note on the use of the BI-hTFR1 capsids from the Deverman Lab:
BI-hTFR1 and the other capsids described in this study bind specifically to the human Transferrin receptor (TfR1) and do not detectably bind the Transferrin receptor from other species. Therefore, users of these plasmids are advised to use the capsids with a suitable humanized TFRC knock-in mouse model or in cells expressing human TfR1. No enhanced tropism should be expected in any other species, including macaques or other species of nonhuman primates.
Please see the accompanying article or contact Dr. Deverman for more information regarding the use of these capsids.
Plasmids from Article
| ID | Plasmid | Purpose |
|---|---|---|
| 218793 | pAAV-CAG-SV40NLSf-mScarletP2A-hLuc | AAV reporter genome plasmid |
| 218794 | pAAV-CMV-CBA-hGBA1-HA-hGHpA | AAV reporter genome plasmid |
| 218795 | pAAV-CMV-CBA-hGBA1-cm-hGBA1-hGHpA | AAV reporter genome plasmid |
| 218796 | kiCAP-AAV-BI-hTFR1 | RepCap for AAV production |
| 218797 | kiCAP-AAV-BI-hTFR1-2 | RepCap for AAV production |
| 218798 | kiCAP-AAV-BI-hTFR1-3 | RepCap for AAV production |
| 218799 | kiCAP-AAV-BI-hTFR1-4 | RepCap for AAV production |