Dual alpha-globin and truncated EPO receptor knockin restores hemoglobin production in alpha-thalassemia-derived red blood cells.
Chu SN, Soupene E, Wienert B, Yin H, Sharma D, McCreary T, Jia K, Homma S, Hampton JP, Gardner JM, Conklin BR, MacKenzie TC, Porteus MH, Cromer MK
bioRxiv [Preprint]. 2024 May 7:2023.09.01.555926. doi: 10.1101/2023.09.01.555926.
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Plasmids from Article
| ID | Plasmid | Purpose |
|---|---|---|
| 232408 | MKC62_HBB(HBA1reg-HBA1-2A-YFP) | AAV production plasmid for HBA1-2A-YFP vector from Fig. 2 that mediates HDR at HBB locus using HBB sg7 gRNA. HBA gene includes HBA1 exons and introns; HBA1 UTRs flank full HBA1-2A-YFP cassette. |
| 232409 | MKC83_HBB(HBBreg-HBA1) | AAV production plasmid for HBB UTRs vector from Fig. 3 that mediates HDR at HBB locus using HBB sg7 gRNA. HBA gene includes HBA1 exons and introns; HBB UTRs flank HBA1 cassette. |
| 232410 | MKC84_HBB(HBA1reg-HBA1) | AAV production plasmid for HBA1 UTRs vector from Fig. 3 that mediates HDR at HBB locus using HBB sg7 gRNA. HBA gene includes HBA1 exons and introns; HBA1 UTRs flank HBA1 cassette. |
| 232411 | MKC106_HBB(HBA1reg-HBA1-IRES-tEPOR) | AAV production plasmid for HBA+tEPOR vector from Figs. 4-6 that mediates HDR at HBB locus using HBB sg7 gRNA. HBA gene includes HBA1 exons and introns; HBA1 UTRs flank HBA+tEPOR cassette. |