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Cell Therapy News
Cell Therapy News is an online resource dedicated to sharing the latest cell therapy, gene therapy, and regenerative medicine research.
In a First, a Gene Therapy Seems to Slow Huntington Disease
[Science] UniQure reported in a press release that its drug reduced the progression of Huntington symptoms by 75% over 3 years in 12 people who had received it versus a comparison group.
CAR T Cell-Mediated Bone Marrow Inflammation Causes Hematotoxicity and Favors Clonal Hematopoiesis
[Science Translational Medicine] The authors used a mouse B cell lymphoma model to investigate the mechanisms underlying hematotoxicity after CAR T cell therapy and found that CAR T cells were required for hematotoxocity.
Gene Therapy and mRNA Drugs Approach for Mitochondrial OXPHOS Deficiencies
[Molecular Therapy] Scientists focus on gene therapy and mRNA drug approaches for nuclear-encoded gene defects causing isolated, combined, or multiple oxidative phosphorylation defects and mitochondrial-encoded gene defects for which a gene replacement approach has been tested, and on the allotopic expression of mtDNA genes.
Oligodendrocyte-Targeted Adeno-Associated Virus Gene Therapy for Canavan Disease in Children: A Phase I/II Trial
[Nature Medicine] This open-label phase I/II clinical study used a novel recombinant vector, rAAV-Olig001, with selective tropism for oligodendrocytes, to deliver gene therapy for Canavan disease.
Enhancing RNA Base Editing on Mammalian Transcripts with Small Nuclear RNAs
[Nature Chemical Biology] Compared with adenosine deaminase acting on RNA (ADAR)-recruiting circular RNAs, researchers found that guided A>I snRNAs consistently increase adenosine-to-inosine editing for higher exon count genes, perturb substantially fewer off-target genes, and localize more persistently to the nucleus where ADAR is expressed.
Chimeric Enzymes Enhance Treatment Potential for Globoid Cell Leukodystrophy through Hematopoietic Stem Cell Gene Therapy
[Molecular Therapy] The authors presented a strategy using lentiviral vector-mediated ex vivo hematopoietic stem/progenitor cell transplantation-gene therapy with a chimeric galactosylceramidase enzyme that incorporates peptides from alpha-L-iduronidase and apolipoprotein E II to enhance expression and blood-brain barrier penetration.
Adipocyte Cell Therapy Targeting Bone Morphogenetic Protein Signaling Alleviates Fibroadipose Tissue Deposition in Secondary Lymphedema
[Molecular Therapy] Investigators identified bone morphogenetic protein ligands (BMPs) as mediators of fibroadipose tissue deposition through in vitro experiments with human lymphedema fluid and BMP-specific inhibitor.
The Endogenous T Cell Landscape Is Reshaped by CAR-T Cell Therapy and Predicts Treatment Response in Multiple Myeloma
[Leukemia] Scientists investigated the co-evolutionary dynamics between CAR-T cells and the immune microenvironment in myeloma patients undergoing anti-BCMA CAR-T cell therapy at single-cell resolution.
Microbiota-Derived Butyrate Potentiates MSLN CAR-T Cell Therapy by Metabolic Reprogramming and Extracellular Matrix Remodeling
[Biomedicine & Pharmacotherapy] In a small patient cohort, preliminary metagenomic and metabolomics data suggested an association between higher butyrate levels and improved CAR-T responses.
A Novel Functional Gene Delivery Platform Based on a Commensal Human Anellovirus Demonstrates Transduction in Multiple Tissue Types
[Molecular Therapy-Methods & Clinical Development] Researchers reported development of a gene delivery vector system based on an anellovirus of the Betatorquevirus genus. Production is enabled by the development of the Self-Amplifying Trans-complementation of a Universal Recombinant aNellovector system.
Lentiviral-Mediated panErbB CAR-T Cell Therapy against Head and Neck Squamous Cell Carcinomas: A Non-Genotoxic Therapy for Patients with Fanconi Anemia
[Molecular Therapy Oncology] Scientists showed that head and neck squamous cell carcinoma cell lines from both the general population and patients with Fanconi anemia express ErbB family members, which can be recognized by the T1E panErbB ligand.
Fibroblast Activation Protein-Targeted CAR-T Cells Induce Apoptosis in Murine Cardiac Myofibroblasts
[Cardiovascular Therapeutics] Using two delivery approaches—lentiviral vectors and lipid nanoparticles—investigators generated FAP-CAR–engineered Jurkat cells as a preliminary screening model and evaluated their CAR expression, target recognition, and in vitro cytotoxic activity.
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.
