Keep Current with the Latest in Cell Biology Research

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.

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