Plasma from Patients with Bacterial Sepsis or Severe COVID-19 Induces Suppressive Myeloid Cell Production from Hematopoietic Progenitors In Vitro

To examine the ontogeny and function of MS1 cells, researchers developed a cellular model for inducing CD14+ MS1 monocytes from healthy bone marrow hematopoietic stem and progenitor cells.
[Science Translational Medicine]
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Development of β-Globin Gene Correction in Human Hematopoietic Stem Cells as a Potential Durable Treatment for Sickle Cell Disease

Researchers demonstrated the preclinical feasibility, efficacy, and toxicology of the β-globin gene correction in plerixafor-mobilized CD34+ cells from healthy and sickle cell disease patient donors.
[Science Translational Medicine]
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CRISPR Mediated Targeting of DUX4 Distal Regulatory Element Represses DUX4 Target Genes Dysregulated in Facioscapulohumeral Muscular Dystrophy

In an immortalized patient myoblast cell line, researchers demonstrated that targeting DUX4 led to substantial downregulation of not only pathogenic DUX4 transcripts, but also a subset of its target genes that are known biomarkers of facioscapulohumeral muscular dystrophy.
[Scientific Reports]
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Modeling Pancreatic Pathophysiology Using Genome Editing of Adult Stem Cell-Derived and Induced Pluripotent Stem Cell (iPSC)-Derived Organoids

Scientists provide highlights on the development of organoid technology, and the use of this culture system to study the pathophysiology of specific mutations in the development of pancreatic and gastric cancer.
[American Journal of Physiology-Gastrointestinal and Liver Physiology]
Sekine, K., Ogawa, S., Tsuzuki, S., Kobayashi, T., Ikeda, K., Nakanishi, N., Takeuchi, K., Kanai, E., Otake, Y., Okamoto, S., Kobayashi, T., Takebe, T., & Taniguchi, H. (2020). Generation of human induced pluripotent stem cell-derived liver buds with chemically defined and animal origin-free media. Scientific Reports, 10(1), 17937. https://doi.org/10.1038/s41598-020-73908-1 Cite
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Plasma from Patients with Bacterial Sepsis or Severe COVID-19 Induces Production of Suppressive Myeloid Cells from Human Hematopoietic Progenitor Cells In Vitro

To examine the ontogeny and function of MS1 cells, scientists developed a cellular model for inducing CD14+ MS1 monocytes by treating human hematopoietic stem and progenitor cells from healthy bone marrow donors in culture with plasma from patients with severe bacterial infection or SARS-CoV-2 infection.
[Science Translational Medicine]
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A Consolidated Aav System for Single Cut CRISPR Correction of a Common Duchenne Muscular Dystrophy Mutation

Scientists devised a single cut gene editing method using a compact Staphylococcus aureus Cas9 to restore the open reading frame of exon 51, the most commonly affected out-of-frame exon in Duchenne muscular dystrophy. Editing of exon 51 in cardiomyocytes revealed a strong preference for exon reframing via a two nucleotide deletion.
[Molecular Therapy-Methods & Clinical Development]
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Generation and Genetic Correction of USH2A c.2299delG Mutation in Patient-Derived Induced Pluripotent Stem Cells

To study the molecular mechanisms of Usher syndrome and develop a gene therapy strategy, researchers generated human iPSCs from peripheral blood mononuclear cells obtained from a patient carrying compound heterozygous variants of USH2A c.2299delG and c.1256G>T and the patient’s healthy sibling.
[Genes]
Liu, X., Lillywhite, J., Zhu, W., Huang, Z., Clark, A. M., Gosstola, N., Maguire, C. T., Dykxhoorn, D., Chen, Z.-Y., & Yang, J. (2021). Generation and Genetic Correction of USH2A c.2299delG Mutation in Patient-Derived Induced Pluripotent Stem Cells. Genes, 12(6), 805. https://doi.org/10.3390/genes12060805 Cite
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Ubiquitin-Specific Protease 3 Deubiquitinates and Stabilizes Oct4 Protein in Human Embryonic Stem Cells

Investigators generated a ubiquitin-specific protease 3 gene knockout using the CRISPR/Cas9 system and demonstrated that USP3 acts as a protein stabilizer of Oct4 by deubiquitinating Oct4.
[International Journal of Molecular Sciences]
Rhie, B.-H., Antao, A. M., Karapurkar, J. K., Kim, M.-S., Jo, W.-J., Ramakrishna, S., & Kim, K.-S. (2021). Ubiquitin-Specific Protease 3 Deubiquitinates and Stabilizes Oct4 Protein in Human Embryonic Stem Cells. International Journal of Molecular Sciences, 22(11), 5584. https://doi.org/10.3390/ijms22115584 Cite
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Targeted Delivery of CRISPR-Cas9 and Transgenes Enables Complex Immune Cell Engineering

The authors developed a one-step strategy using engineered lentiviral particles to introduce Cas9 RNPs and a CAR transgene into primary human T cells without electroporation.
[Cell Reports]
Hamilton, J. R., Tsuchida, C. A., Nguyen, D. N., Shy, B. R., McGarrigle, E. R., Espinoza, C. R. S., Carr, D., Blaeschke, F., Marson, A., & Doudna, J. A. (2021). Targeted delivery of CRISPR-Cas9 and transgenes enables complex immune cell engineering. Cell Reports, 35(9). https://doi.org/10.1016/j.celrep.2021.109207 Cite
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Small Leucine Zipper Protein Promotes the Metastasis of Castration-Resistant Prostate Cancer through Transcriptional Regulation of Matrix Metalloproteinase-13

The authors investigated the role of small leucine zipper protein in matrix metalloproteinases -13 expression and its involvement in castration-resistant prostate cancer metastasis.
[Carcinogenesis]
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Acid Ceramidase Controls Apoptosis and Increases Autophagy in Human Melanoma Cells Treated with Doxorubicin

Investigators found that doxorubicin activates the autophagic response in wild-type A375 cells, which effectively resist apoptotic cell death.
[Scientific Reports]
Lai, M., Amato, R., La Rocca, V., Bilgin, M., Freer, G., Spezia, P., Quaranta, P., Piomelli, D., & Pistello, M. (2021). Acid ceramidase controls apoptosis and increases autophagy in human melanoma cells treated with doxorubicin. Scientific Reports, 11(1), 11221. https://doi.org/10.1038/s41598-021-90219-1 Cite
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