Researchers described an approach to edit the recombination-activating gene 2 (RAG2) locus for therapeutic applications.
By Tarun Sai LomteNov 1 2023Reviewed by Danielle Ellis, B.Sc. In a recent study published in Nature Communications, researchers described an approach to edit the recombination-activating gene 2 locus for therapeutic applications.
Nevertheless, human leucocyte antigen -matched donors are rare, and haploidentical HSCT, the alternative therapy, reduces survival rate to 60% to 70%. An ideal alternative would be ex vivo manipulation of the patient’s hematopoietic stem and progenitor cells and subsequent transfer to the patient as autologous HSCT.
The first donor used 400 bp homology arms flanking the Cas9 cut site for insertion. In contrast, the second donor used a 400 bp left homology arm covering the sequence upstream of the cut site and a 400 bp right homology arm spanning the sequence downstream of the RAG2 stop codon to replace the whole RAG2 CDS with the donor DNA .
Related StoriesBesides, two additional CDSR correction donors with synthetic regulatory elements following the tNGFR sequence were synthesized. One contained the WPRE-BGHpA sequences, and the other had the BGHpA sequence. The correction donors showed effective locus-specific HDR. In-out polymerase chain reaction results corroborated the highly effective HDR.
Indeed, endogenous RAG2 expression was eliminated in KI-KO cells, but dcoRAG2 cDNA expression was robust. Moreover, dcoRAG2 transgene expression was still tightly regulated and was not overexpressed, facilitating the development of T cells by the differentiation of engineered cells into CD3+ T cells by day 28.
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