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Engineered Allogeneic Stem Cells Orchestrate T Lymphocyte Driven Immunotherapy in Immunosuppressive Leptomeningeal Brain Metastasis

January 24, 2025

[JNCI-Journal of The National Cancer Institute] A paired matched RNA expression dataset of patients with non-small cell lung cancers and brain metastases (BMs) was analyzed to idenfiy BM specific suppressive tumor microenvironment features.

Neonatal but not Juvenile Gene Therapy Reduces Seizures and Prolongs Lifespan in SCN1B-Dravet Syndrome Mice

January 24, 2025

[Journal of Clinical Investigation] The authors developed a proof-of-principle gene replacement strategy for developmental and epileptic encephalopathy (DEE) 52. They tested an adeno-associated viral vector encoding β1 subunit cDNA in Scn1b null mice.

Branched Endosomal Disruptor (BEND) Lipids Mediate Delivery of mRNA and CRISPR-Cas9 Ribonucleoprotein Complex for Hepatic Gene Editing and T Cell Engineering

January 24, 2025

[Nature Communications] Scientists developed a platform for synthesizing a class of branched ionizable lipids that improved endosomal escape.

In Vivo CRISPR–Cas9 Genome Editing in Mice Identifies Genetic Modifiers of Somatic CAG Repeat Instability in Huntington’s Disease

January 23, 2025

[Nature Genetics] To identify novel modifiers of somatic expansion, the authors used CRISPR–Cas9 editing in Huntington’s disease knock-in mice to enable in vivo screening of expansion-modifier candidates at scale.

Microenvironment Actuated CAR T Cells Improve Solid Tumor Efficacy Without Toxicity

January 23, 2025

[Science Advances] To improve the selectivity of the CAR T cell, scientists engineered a synthetic Notch receptor that selectively expressed the CAR upon binding to P-selectin, a cell adhesion protein overexpressed in tumor neovasculature.

Impact of TP53 Alteration on Allogeneic Hematopoietic Cell Transplantation Outcomes for Myelodysplastic Syndromes

January 22, 2025

[Bone Marrow Transplantation] Scientists revealed the role of TP53 alterations in the prognosis of myelodysplastic syndrome (MDS) patients undergoing allogeneic hematopoietic stem cell transplantation.

The Maternal X Chromosome Affects Cognition and Brain Aging in Female Mice

January 22, 2025

[Nature] The authors tested whether skew towards an active maternal X (Xm) chromosome influenced the brain and body—and then delineated unique features of Xm neurons and paternal X neurons.

Triple Knockdown of CD11a, CD49d, and PSGL1 in T Cells Reduces CAR-T Cell Toxicity but Preserves Activity against Solid Tumors in Mice

January 22, 2025

[Science Translational Medicine] Researchers demonstrated that targeting the cell adhesion and migration molecules lymphocyte function–associated antigen 1 and very late activation antigen 4 with blocking antibodies reduced the on-target, off-tumor toxicity of CAR T cells in mice.

CD133+PD-L1+ Cancer Cells Confer Resistance to Adoptively Transferred Engineered Macrophage-Based Therapy in Melanoma

January 21, 2025

[Nature Communications] Researchers discovered a subpopulation of cancer cells with upregulated CD133 and programmed death-ligand 1 in mouse melanoma, resistant to the phagocytosis by the transferred macrophages.

Long-Term Safety of Lentiviral or Gammaretroviral Gene-Modified T Cell Therapies

January 21, 2025

[Nature Medicine] The authors evaluated safety outcomes in 783 patients over more than 2,200 total patient-years of observation from 38 T cell therapy trials.

Glut3 Overexpression Improves Environmental Glucose Uptake and Antitumor Efficacy of CAR-T Cells in Solid Tumors

January 20, 2025

[Journal for ImmunoTherapy of Cancer] Glucose transporters were detected and overexpressed in CAR-T cells. The impacts of glucose restriction on CAR-T cells were checked in vitro and in vivo.

PRDM1 Is a Key Regulator of the Natural Killer T–Cell Central Memory Program and Effector Function

January 20, 2025

[Cancer Immunology Research] To find regulators of natural killer T cells functional fitness, investigators developed a CRISPR/Cas9-based mutagenesis screen that employed a guide RNA library targeting 1,118 immune-related genes.

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.