 | | Vol. 23.31 – 12 September, 2022 |
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| Researchers successfully developed a two-in-one approach to generate non-viral, gene-specific targeted CAR-T cells through CRISPR–Cas9. Using the optimized protocol, they demonstrated feasibility in a preclinical study by inserting an anti-CD19 CAR cassette into the AAVS1 safe-harbor locus.
[Nature] |
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 | | PUBLICATIONSRanked by the impact factor of the journal |
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| Scientists showed that human neural progenitor cells transduced with glial cell line-derived neurotrophic factor (GDNF) differentiated to astrocytes protected spinal motor neurons and were safe in animal models.
[Nature Medicine] |
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| Investigators described the tissue engineering of human thymus organoids from inducible PSCs that could support the de novo generation of a diverse population of functional human T cells.
[Nature Methods] |
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| The authors developed a fully humanized immunocompetent model of HER2+ breast cancer recapitulating ex vivo the biological processes that associated with patients’ response to treatment.
[Nature Communications] |
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| Investigators generated mouse and human mRNA-based multifunctional T cells co-expressing a multitargeting CAR based on the NKG2D receptor and the pro-inflammatory cytokines IL12 and IFNa2 and assessed their anti-glioma activity in vitro and in vivo.
[Clinical Cancer Research] |
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| Researchers showed fratricide of CD7 CAR T-cells could be fully prevented using ibrutinib and dasatinib, the pharmacologic inhibitors of key CAR/CD3ζ signaling kinases.
[Molecular Therapy] |
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| Scientists assessed the mechanisms of how intrahepatic co-infusion of human alpha-1 antitrypsin-engineered mesenchymal stromal cells improved survival of human islet grafts post-transplantation.
[Diabetes] |
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| In vivo evaluations were carried out on a subcutaneous implantation in rats, and the results demonstrated the importance of the local oxygen availability in a series of crucial events for tissue regeneration.
[Biomaterials Advances] |
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| Researchers developed a novel optical sensor system, instead of a conventional Clark-type needle probe, for measuring tissue oxygen partial pressure (pO2) and found that pO2 of the subcutaneous space was extremely low in comparison to other sites.
[Scientific Reports] |
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| The authors describe the state of the art in β-cell replacement with a focus on its efficacy and clinical benefits, on the actual limitations and still unmet needs, and on the latest findings and future directions.
[Stem Cells Translational Medicine] |
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| Scientists summarize the current state of animal models in the CAR-T cell therapy field, and the advantages and disadvantages of each approach depending on the preclinical questions being asked.
[Molecular Therapy-Methods & Clinical Development] |
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| University and industry leaders are celebrating an agreement to form a 30-year strategic partnership between the University of Pittsburgh (Pitt) and biotech company ElevateBio to accelerate the development of highly innovative cell and gene therapies at Pitt BioForge.
[University of Pittsburgh] |
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| Immusoft, a clinical-stage cell therapy company dedicated to improving the lives of patients with rare diseases, announced that the US FDA has cleared the Investigational New Drug Application (IND) for ISP-001 in the treatment of Mucopolysaccharidosis type I (MPS I).
[Immusoft] |
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| At the National Institutes of Health, a surgical team successfully implanted a patch of tissue made from patient cells with the goal of treating advanced “dry” age-related macular degeneration (AMD), also known as geographic atrophy.
[National Institutes of Health] |
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| | Max Delbrück Center for Molecular Medicine – Berlin, Germany |
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| | Lewis Katz School of Medicine, Temple University – Philadelphia, Pennsylvania, United States |
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| | European Molecular Biology Laboratory – Heidelberg, Germany |
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| | German Cancer Research Center in the Helmholtz Association – Heidelberg, Germany |
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| | University of Copenhagen – Copenhagen, Denmark |
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