Cell Therapy News
Cell Therapy News is an online resource dedicated to sharing the latest cell therapy, gene therapy, and regenerative medicine research.
Transient mRNA CAR T Cells Targeting GD2 Provide Dose-Adjusted Efficacy against Diffuse Midline Glioma and High Grade Glioma Models
[Neuro-Oncology] Researchers evaluated transient CAR T cells targeting GD2 created with mRNA, assessing for efficacy and safety in cell line, organoid, and in vivo xenograft models with repetitive intratumoral dosing.
An Engineered U7 Small Nuclear RNA Scaffold Greatly Increases ADAR-Mediated Programmable RNA Base Editing
[Nature Communications] An optimized scaffold with a stronger synthetic U7 promoter enables 76% RNA editing in vitro from a single DNA construct per cell, and 75% editing in a Hurler syndrome mouse brain after one systemic AAV injection, surpassing circular gRNA approaches.
Lentiviral Gene Therapy with Reduced-Intensity Conditioning for Sickle Cell Disease: A Phase I/II Trial
[Nature Medicine] Investigators reported results of seven patients treated in a first-in-human phase I/II study for sickle cell disease using reduced-intensity conditioning transplant of autologous hematopoietic stem cells genetically modified with a lentiviral vector, with 2–7 yr of follow-up.
CD97-Directed CAR-T Cells with Enhanced Persistence Eradicate Acute Myeloid Leukemia in Diverse Xenograft Models
[Cell Reports Medicine] Researchers proposed CD97 as a potential target for CAR-T therapy against acute myeloid leukemia (AML) based on its broader and higher expression on AML cells compared to normal hematopoietic stem and progenitor cells.
Professor Leo Chou Receives Ontario Early Researcher Award to Advance Vaccine and Immunotherapy Delivery
[U of T Engineering] Professor Leo Chou has received an Early Researcher Award from the Government of Ontario to support his work on improving vaccine and immunotherapy delivery using DNA-based nanomaterials.
Enhancing the Potency of In Vivo Lentiviral Vector Mediated Gene Therapy to Hepatocytes
[Nature Communications] The authors showed that the a posteriori enhancement increased transgene output without expansion of hepatocytes bearing lentiviral vector genomic integrations near cancer genes, in mouse models of hemophilia, an inherited coagulation disorder.
Gene Therapy for Cardiac Arrhythmias
[Nature Reviews Cardiology] Investigators describe the rationale for gene therapy to treat cardiac arrhythmias and discuss advantages and disadvantages of gene silencing, gene replacement, gene suppression-and-replacement and gene editing technologies.
A Collagenase Nanogel Backpack Improves CAR-T Cell Therapy Outcomes in Pancreatic Cancer
[Nature Nanotechnology] Scientists showed that an approach based on collagenase nanogel generally improves the outcome of T cell-based therapies, and specifically of CAR-T cell therapy.
Programmable Control of Spatial Transcriptome in Live Cells and Neurons
[Nature] Researchers presented CRISPR-mediated transcriptome organization, a system that harnesses RNA-guided, nuclease-dead dCas13 for programmable control of endogenous RNA localization in live cells.
Cell Trajectory Modulation: Rapid Microfluidic Biophysical Profiling of CAR T Cell Functional Phenotypes
[Nature Communications] Investigators introduced the Cell Trajectory Modulation assay, a microfluidic, label-free approach for the rapid evaluation of the functional attributes of CAR T cells based on biophysical features.
Gene Therapy Prevents Onset of Mitochondrial Cardiomyopathy in Neonatal Mice with Ndufs6 Deficiency
[Cell Death Discovery] Researchers determined whether adeno-associated virus 9-based gene therapy could prevent or rescue Ndufs6 deficiency-induced mitochondrial cardiomyopathy at different disease stages.
CRISPR-Cas Therapies Targeting Bacteria
[Nature Reviews Bioengineering] Investigators explore how the CRISPR–Cas toolbox can be engineered to kill or modify specific bacteria.
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.