Table 1.
Overview of the current developments on RAG gene therapy.
| RAG1/2-SCID | Gene therapy strategy | Vector/endonuclease | Stage |
|---|---|---|---|
| RAG1 | Gene addition | MLV/RV | In vivo mice (194) |
| RAG1 | Gene addition | LV | In vivo mice (109) |
| RAG1 | Gene addition | LV | In vivo mice (199) |
| RAG1 | Gene addition | LV | Phase I/II (108, 112) |
| RAG2 | Gene addition | MLV/RV | In vivo mice (201) |
| RAG2 | Gene addition | LV | In vivo mice (202) |
| RAG2 | Gene addition | LV | In vivo mice (203) |
| RAG2 | Gene editing | CRISPR-Cas9/rAAV6 | In vitro iPSCs (204) |
| RAG2 | Gene editing | CRISPR-Cas9/rAAV6 | In vitro HSPCs (205) |
| RAG2 | Gene editing | CRISPR-Cas9/AAV6 | In vitro HSPCs (206) |
MLV, Moloney leukemia virus; RV, retro virus; LV, lentivirus; rAAV6, recombinant adeno-associated virus serotype 6; HSPCs, hematopoietic stem and progenitor cells; iPSCs, induced pluripotent stem cells.