Gene replacement of alpha-globin with beta-globin restores hemoglobin balance in beta-thalassemia-derived hematopoietic stem and progenitor cells.
Cromer MK, Camarena J, Martin RM, Lesch BJ, Vakulskas CA, Bode NM, Kurgan G, Collingwood MA, Rettig GR, Behlke MA, Lemgart VT, Zhang Y, Goyal A, Zhao F, Ponce E, Srifa W, Bak RO, Uchida N, Majeti R, Sheehan VA, Tisdale JF, Dever DP, Porteus MH
Nat Med. 2021 Apr;27(4):677-687. doi: 10.1038/s41591-021-01284-y. Epub 2021 Mar 18.
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Plasmids from Article
| ID | Plasmid | Purpose |
|---|---|---|
| 232401 | KRD2_HBA1(HBA1reg-UbC-GFP) | AAV production plasmid for HBA1 WGR vector from Fig. 1 that mediates HDR at HBA1 locus using HBA1 sg5 gRNA. HBA1 UTRs flank UbC-GFP cassette; GFP is followed by BGH polyA. HAs are ~400bp each. |
| 232402 | KRD9_HBA1(HBA1reg-HBB) | AAV production plasmid for HBA1 UTRs vector from Fig. 3 that mediates HDR at HBA1 locus using HBA1 sg5 gRNA. HBB gene includes introns; HBA1 UTRs flank HBA1 cassette. HAs are ~400bp each. |
| 232403 | KRD22_HBA1(HBA1reg-HBB-longHAs) | AAV production plasmid for HBA1 long HAs vector from Figs. 3-6 that mediates HDR at HBA1 locus using HBA1 sg5 gRNA. HBB gene includes introns; HBA1 UTRs flank full HBA1-2A-YFP cassette. HAs are ~900bp |