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lbeveridge

Characterization of RNA Editing and Gene Therapy with a Compact CRISPR-Cas13 in the Retina

[Proceedings of The National Academy of Sciences of The United States of America] Investigators reported significant transcriptomic signatures of Cas13bt3 expression in retinal cells and show all-in-one adeno-associated virus gene therapy with Cas13bt3 can effectively silence VEGFA mRNA in human retinal organoids and humanized VEGF transgenic mouse models.

FDA Grants Accelerated Approval to Asciminib for Adult Patients With Newly Diagnosed Ph+ CML-CP

[Pharmacy Times] The FDA granted an accelerated approval to asciminib for adult patients with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase.

A Mutant BCL11B-N440K Protein Interferes with BCL11A Function During T Lymphocyte and Neuronal Development

[Nature Immunology] Investigators showed that mice harboring the corresponding Bcl11bN440K mutation show the emergence of natural killer/group 1 innate lymphoid cell-like NKp46+ cells in the thymus and reduction in TBR1+ neurons in the neocortex, which were observed with loss of Bcl11a but not Bcl11b.

Self-Assembled Aldehyde Dehydrogenase-Activatable Nano-Prodrug for Cancer Stem Cell-Enriched Tumor Detection and Treatment

[Nature Communications] Scientists reported a self-assembled nano-prodrug composed of aldehyde dehydrogenases activatable photosensitizer and disulfide-linked all-trans retinoic acid for diagnosis and targeted treatment of CSCs enriched tumors.

Polyclonality Overcomes Fitness Barriers in Apc-Driven Tumorigenesis

Investigators combined multicolor lineage tracing with chemical mutagenesis in mice to show that a large proportion of intestinal tumours have a multiancestral origin. Polyclonal tumors retained a structure comprising subclones with...

Contemporary Concise Review 2023: Interstitial Lung Disease

Crosstalk between vascular endothelial cells and fibroblasts in Idiopathic Pulmonary Fibrosis mediated by bone morphogenic protein signalling may be important for remodeling of the lung. A novel modality for monitoring...

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Microenvironmental Cell Interactions Are Essential for Sustaining Functionality of Myelofibrosis Malignant Stem Cells

[Blood] To model in vivo cancer stem cell/tumor microenvironment interactions and elucidate their functional consequences, scientists focused on myelofibrosis, a stem cell driven myeloproliferative neoplasm.

Mechanometabolism Instructs Hematopoietic Stem Cell Specification

[Journal of Experimental Medicine] Researchers reported that shear stress triggers adaptation in mitochondrial composition, ultrastructure, and function, which are essential for hematopoietic fate and engraftment potential.

Ribosomal Protein Control of Hematopoietic Stem Cell Transformation through Regulation of Metabolism

[Cell Reports] The authors reported that expression of the ribosomal protein RPL22 is frequently reduced in human myelodysplastic syndrome and AML, and reduced RPL22 expression is associated with worse outcomes.