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Cell Therapy News is an online resource dedicated to sharing the latest cell therapy, gene therapy, and regenerative medicine research.
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CD19 CAR T-Cell Therapy for Autoimmune Hemolytic Anemia
[New England Journal of Medicine] Investigators enrolled patients from a compassionate-use program and those from a Phase I study who had primary multirefractory autoimmune hemolytic anemia. Each patient received a single infusion of autologous CD19 CAR T cells.
CD4+ T Cells Mediate CAR-T Cell-Associated Immune-Related Adverse Events after BCMA CAR-T Cell Therapy
[Nature Medicine] Among 198 patients treated with ciltacabtagene autoleucel or idecabtagene vicleucel, 27 developed CAR T cell therapy-associated immune-related adverse events (CirAEs). This included one remarkable case with three distinct CirAEs in association with an extreme CD4+ CAR T cell expansion.
Contaminating Plasmid Sequences and Disrupted Vector Genomes in the Liver following Adeno-Associated Virus Gene Therapy
[Nature Medicine] Scientists conducted a case study of a child with spinal muscular atrophy type 1 experiencing substantial hepatitis after receiving onasemnogene abeparvovec, undertaking long- and short-read metagenomic sequencing of liver tissue.
An Engineered UGA Suppressor tRNA Gene for Disease-Agnostic AAV Delivery
[Nature Biotechnology] The authors demonstrated that an engineered UGA-sup-tRNA gene, designed with transcriptional regulatory elements, can be efficiently packaged into recombinant AAV for in vivo delivery.
BACH2 Dosage Establishes the Hierarchy of Stemness and Fine-Tunes Antitumor Immunity in CAR T Cells
[Nature Immunology] Investigators showed that BACH2 dose-dependently regulates long-term stem-like differentiation and antitumor immunity of CAR T cells.
BACH2 Regulates T Cell Lineage State to Enhance CAR T Cell Function
[Nature Immunology] Researchers showed that 41BB induces BACH2, a transcriptional regulator that directs stem and memory programs.
Single-Cell Transcriptomics of Multi-Site Cell Therapy in Osteoarthritis: Tissue-Specific Treatment Correlations
[Molecular Therapy] The authors used single-cell RNA sequencing to analyze bone marrow aspirate concentrate and stromal vascular fraction samples in a clinical trial of autologous cell therapies.
Mutant Calreticulin Enables Potent and Selective CAR-T Cell Therapy in Preclinical Models of Myeloproliferative Neoplasms
[Journal of Immunotherapy of Cancer] Five distinct CARs were designed based on available monoclonal antibody sequences that target mutant calreticulin and were subsequently used to generate CAR-T cells.
Impact of Antiplatelet Therapy on the Hemostatic Efficacy of Platelet-Targeted FVIII Gene Therapy in Hemophilia a Mice
[Blood Advances] Researchers investigated the effect of antiplatelet agents on the efficacy of platelet-FVIII in HA mice. 2bF8Tg mice express FVIII in platelets controlled by the αIIb promoter and were treated with aspirin, clopidogrel, or the αIIbβ3-blocking antibody Leo.H4.
Mapping Clinical CAR-T Cells: Insights from scRNA-Seq
[Trends in Molecular Medicine] The authors provide a comprehensive summary of published studies that have used scRNA-seq to analyze clinical CAR-T cells, focusing on T cell exhaustion, cytotoxicity, memory, expansion, clonal diversity, and cytokines.
Cell Therapy in Sarcoma: Current Landscape and Future Directions
[Journal of Immunotherapy of Cancer] Scientists provide the current landscape and growing potential of cell therapies in sarcomas, including TCR-T, CAR-T cells, tumor-infiltrating lymphocytes, natural killer cells, and mesenchymal stromal cells.
$1 Million Grant from Blood Cancer United to Escalate Groundbreaking CAR T-Cell Therapy Study
[University of Colorado Anschutz] A research team from Gates Institute at University of Colorado Anschutz has been awarded $1 million from Blood Cancer United® to advance a new immunotherapy approach for patients with treatment-resistant acute myeloid leukemia.
Published since 2002, Cell Therapy News is our longest running online publication. Our editorial team curates the top publications, reviews, and news on in vivo gene therapy, cell therapy, and immunotherapy research. With over 15,000 subscribers and followers across our website, email newsletter, and Twitter feed, we are proud to facilitate the communication of cutting-edge cell therapy innovations.

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