B2M knockout cord blood-derived megakaryocytes + platelets

B2M knockout cord blood-derived megakaryocytes + platelets are an experimental, engineered cell and cell-derived product generated ex vivo from human umbilical cord blood CD34+ hematopoietic stem and progenitor cells that have been edited by CRISPR–Cas9 to disrupt the β2-microglobulin (B2M) gene, thereby eliminating surface HLA class I expression on megakaryocytes and their released platelets.[1][4][3] In optimized culture systems using mesenchymal stromal cell co-culture, thrombopoietic cytokines, and Rho-associated kinase (ROCK) inhibition, these edited progenitors are expanded and differentiated into mature megakaryocytes that produce functional platelets with preserved aggregation and degranulation responses while exhibiting markedly reduced susceptibility to killing by allogeneic T cells due to their HLA class I–negative phenotype.[1][4][6] The concept is being developed as an off-the-shelf, adult donor–independent universal platelet source intended primarily for platelet transfusion support, particularly in patients with alloimmune platelet refractoriness or other settings where HLA incompatibility limits conventional platelet transfusion.[1][4][6]

Other names

B2M KO cord blood-derived megakaryocytes + plateletsHLA class I-deficient cord blood-derived megakaryocytes + plateletsBeta-2 microglobulin knockout cord blood-derived megakaryocytes + platelets