CRISPR-Cas9

[Cell Death & Disease] Scientists show that combining TKI treatment with the small-molecule MCL1 inhibitor S63845 exerted strong synergistic antiviability and proapoptotic effects on chronic myeloid leukemia (CML) lines and CD34+ stem/progenitor cells isolated from untreated CML patients in chronic phase.

[Oncogene] Scientists investigated the loss of SOX4 in mammary tumor development utilizing organoids derived from a PyMT genetic mouse model of breast cancer.

[STAR Protocols] Researchers developed erythroblasts with custom phenotypes using CRISPR/Cas9 gene-editing of a human iPSC parent line for the blood group system genes: RHAG, GYPB and XK.

[Fluids and Barriers of the CNS] Investigators applied a novel bioengineering approach to generate human blood-brain barrier organoids by the self-assembly of astrocytes, pericytes and brain endothelial cells with unprecedented throughput and reproducibility using micro patterned hydrogels.

[Frontiers in Immunology] Scientists described an anti-HLA-A2 CAR generated by grafting the complementarity-determining regions of a human monoclonal anti-HLA-A2 antibody into the framework regions of the Herceptin 4D5 single-chain variable fragment and fusing it with a CD28-ζ signaling domain.

[Translational Psychiatry] Spinocerebellar ataxia type 3 (SCA3)-iPSCs could be corrected by the replacement of the abnormal CAG expansions with normal repeats using CRISPR/Cas9-mediated homologous recombination strategy. Corrected SCA3-iPSCs retained pluripotent and normal karyotype, which could be differentiated into a neural stem cell and neuronal cells.