mRNA-Engineered Mesenchymal Stromal Cells Expressing CXCR2 Enhances Cell Migration and Improves Recovery in Inflammatory Bowel Disease

A new strategy to modify MSCs to express specific chemokine receptors using mRNA engineering was developed to evaluate the homing ability of MSCs and its therapeutic effects for inflammatory bowel disease.
[Molecular Therapy-Nucleic Acids]
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Pulmonary Transplantation of Alpha-1 Antitrypsin (AAT)-Transgenic Macrophages Provides a Source of Functional Human AAT In Vivo

The authors investigated the pulmonary transplantation of macrophages transgenic for alpha-1 antitrypsin (AAT) as a potential therapy for AAT deficiency-associated lung pathology.
[Gene Therapy]
Janosz, E., Hetzel, M., Spielmann, H., Tumpara, S., Rossdam, C., Schwabbauer, M., Kloos, D., von Kaisenberg, C., Schambach, A., Buettner, F. F. R., Janciauskiene, S., Lachmann, N., & Moritz, T. (2021). Pulmonary transplantation of alpha-1 antitrypsin (AAT)-transgenic macrophages provides a source of functional human AAT in vivo. Gene Therapy, 1–17. https://doi.org/10.1038/s41434-021-00269-3 Cite
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Distinct Effects of V617F and exon12-Mutated JAK2 Expressions on Erythropoiesis in a Human Induced Pluripotent Stem Cell (iPSC)-Based Model

The iPSCs were used to model JAK2-mutated MPNs. Normal iPSCs underwent lentiviral transduction to overexpress JAK2p.V617F or JAK2p.N542_E543del under a doxycycline-inducible system. The modified iPSCs were differentiated into erythroid cells.
[Scientific Reports]
Nilsri, N., Jangprasert, P., Pawinwongchai, J., Israsena, N., & Rojnuckarin, P. (2021). Distinct effects of V617F and exon12-mutated JAK2 expressions on erythropoiesis in a human induced pluripotent stem cell (iPSC)-based model. Scientific Reports, 11(1), 5255. https://doi.org/10.1038/s41598-021-83895-6 Cite
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Cutaneous and Hepatic Vascular Lesions Due to a Recurrent Somatic GJA4 Mutation Reveal a Pathway for Vascular Malformation

The authors found that the mutant induced changes in cell morphology and activated SGK1, a serine/threonine kinase known to regulate cell proliferation and apoptosis, via non-canonical activation.
[HGG Advances]
Ugwu, N., Atzmony, L., Ellis, K., Panse, G., Jain, D., Ko, C. J., Nassiri, N., & Choate, K. A. (2021). Cutaneous and hepatic vascular lesions due to a recurrent somatic GJA4 mutation reveal a pathway for vascular malformation. Human Genetics and Genomics Advances, 0(0). https://doi.org/10.1016/j.xhgg.2021.100028 Cite
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Genome-Wide CRISPR/Cas9-Knockout in Human Induced Pluripotent Stem Cell (iPSC)-Derived Macrophages

Scientists present an efficient, lentiviral transduction protocol for delivery of CRISPR/Cas9 to macrophages derived from human iPSC with efficiencies close to 100%.
[Scientific Reports]

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Alpha-Enolase (ENO1), Identified as an Antigen to Monoclonal Antibody 12C7, Promotes the Self-Renewal and Malignant Phenotype of Lung Cancer Stem Cells by AMPK/mTOR Pathway

Immunofluorescence was used for antigen localization. After targeted antigen purification by electrophoresis and immunoblot, the antigen was identified by LC-MALDI-TOF/TOF mass spectrometry, immunofluorescence, and immunoprecipitation.
[Stem Cell Research & Therapy]
Shu, X., Cao, K.-Y., Liu, H.-Q., Yu, L., Sun, L.-X., Yang, Z.-H., Wu, C.-A., & Ran, Y.-L. (2021). Alpha-enolase (ENO1), identified as an antigen to monoclonal antibody 12C7, promotes the self-renewal and malignant phenotype of lung cancer stem cells by AMPK/mTOR pathway. Stem Cell Research & Therapy, 12(1), 119. https://doi.org/10.1186/s13287-021-02160-9 Cite
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CD10 Marks Non-Canonical PPARγ-Independent Adipocyte Maturation and Browning Potential of Adipose-Derived Stem Cells

Investigators identified intrinsic CD10 levels as a positive determinant of adipocyte maturation as well as browning potential of human adipose-derived stem cells.
[Stem Cell Research & Therapy]

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Hematopoietic-Stem-Cell-Targeted Gene-Addition and Gene-Editing Strategies for β-hemoglobinopathies

State-of-the-art gene-editing technology has made it possible to repair the β-globin mutation in patient hematopoietic stem cells or target genetic loci associated with reactivation of endogenous γ-globin expression.
[Cell Stem Cell]
Drysdale, C. M., Nassehi, T., Gamer, J., Yapundich, M., Tisdale, J. F., & Uchida, N. (2021). Hematopoietic-Stem-Cell-Targeted Gene-Addition and Gene-Editing Strategies for β-hemoglobinopathies. Cell Stem Cell, 28(2), 191–208. https://doi.org/10.1016/j.stem.2021.01.001 Cite
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Combined Lentiviral- and RNA-Mediated CRISPR/Cas9 Delivery for Efficient and Traceable Gene Editing in Human Hematopoietic Stem and Progenitor Cells

Researchers developed a combinatorial system using stable lentiviral delivery of single guide RNA followed by transient transfection of Cas9 mRNA by electroporation in human cord blood-derived CD34+ hematopoietic stem and progenitor cells.
[Scientific Reports]
Yudovich, D., Bäckström, A., Schmiderer, L., Žemaitis, K., Subramaniam, A., & Larsson, J. (2020). Combined lentiviral- and RNA-mediated CRISPR/Cas9 delivery for efficient and traceable gene editing in human hematopoietic stem and progenitor cells. Scientific Reports, 10(1), 22393. https://doi.org/10.1038/s41598-020-79724-x Cite
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Secretory Cells Dominate Airway CFTR Expression and Function in Human Airway Superficial Epithelia

Researchers identified the cell types that contribute to CFTR expression and function within the proximal-distal axis of the normal human lung. Single cell RNA-seq was performed on freshly isolated human large and small airway epithelial cells.
[American Journal of Respiratory and Critical Care Medicine]
Okuda, K., Dang, H., Kobayashi, Y., Carraro, G., Nakano, S., Chen, G., Kato, T., Asakura, T., Gilmore, R. C., Morton, L. C., Lee, R. E., Mascenik, T., Yin, W.-N., Barbosa Cardenas, S. M., O’Neal, Y. K., Minnick, C. E., Chua, M., Quinney, N. L., Gentzsch, M., … Boucher, R. C. (2020). Secretory Cells Dominate Airway CFTR Expression and Function in Human Airway Superficial Epithelia. American Journal of Respiratory and Critical Care Medicine. https://doi.org/10.1164/rccm.202008-3198OC Cite
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