Cell Therapy News

Cell Therapy News is an online resource dedicated to sharing the latest cell therapy, gene therapy, and regenerative medicine research.

Efficient and Traceless Aptamer-Based Serial Selection of Naïve and Early Memory CD8 T Cells for CAR T Cell Therapy

[Advanced Healthcare Materials] A high-affinity CD62L aptamer and a traceless, sequential selection system were reported for the high-yield and high-purity isolation of CD62LCD8 T cells without residual selection labels.

Evaluation and Prediction of Guide RNA Activities in Genome-Editing Tools

[Nature Reviews Bioengineering] The authors reviewed recent developments in high-throughput evaluations and machine learning-based predictions of genome-editing efficiencies and/or off-target effects, together with recent advances in diverse genome-editing tools.

Tissue-Specific mRNA Delivery and Prime Editing with Peptide-Ionizable Lipid Nanoparticles

[Nature Materials] Scientists reported the rational design of peptide ionizable lipids to assemble lipid nanoparticles with organ-selective mRNA delivery.

Pan-Carcinoma Sialyl-Tn-Targeting Expands CAR Therapy to Solid Tumors

[Cell Reports Medicine] Researchers described AM52.1, a monoclonal antibody with unprecedented specificity for sialyl-Tn and lack of reactivity with healthy tissues.

Retinal Gene Therapy Using Epiretinal AAV-Containing Fibrin Hydrogel Implants

[Science Advances] The authors manufactured high-concentration fibrin hydrogels encapsulating AAV2–green fluorescent protein. Gels had homogeneous AAV distribution, desired mechanical properties, and retained infectivity.

Evaluating CAR-T Cells from Neurofibromatosis Type 1 (NF1) Patients for Targeting AXL in Malignant Peripheral Nerve Sheath Tumors Associated with NF1

[British Journal of Cancer] AXL-CAR-T cells, containing an anti-AXL single-chain variable fragment, were derived from neurofibromatosis type 1 (NF1) patients and healthy donors

CAR T Cell Engineering Impacts Antigen-Independent Activation and Co-Inhibition

[Molecular Therapy-Methods & Clinical Development] Investigators demonstrated that their lentivirally manufactured R110-CAR T cells, targeting a leukemia neoepitope, can also be engineered using non-viral Sleeping Beauty transposition with minimal-sized DNA vectors.

Comparative Analysis of Cell-Specific Promoters in AAV9-Mediated Gene Therapy Targeting the Central Nervous System

[Molecular Therapy-Methods & Clinical Development] Scientists introduced a novel, astrocyte-specific, truncated glial fibrillary acidic protein promoter, named gfaABCD1405, which enhances astrocyte specificity while reducing size, improving utility for gene therapies requiring larger transgenes.

Clinical Gene Therapy Restores Hearing: A Paradigm Shift

[Trends in Molecular Medicine] Investigators synthesize progress in OTOF-related clinical trials, highlighting translational foci such as inner ear drug delivery, trial design, safety assessments, and auditory restoration outcomes.

MD Anderson and Phoenix SENOLYTIX Announce Strategic Cross-Licensing Agreement to Enhance Inducible Switch Technologies for Cell and Gene Therapies

[University of Texas MD Anderson Cancer Center] The University of Texas MD Anderson Cancer Center and Phoenix SENOLYTIX, Inc., announced a global cross-licensing agreement to facilitate work that will further enhance the development of inducible switch technologies for use in cell and gene therapies.

Development of an AAV-Delivered microRNA Gene Therapy for Myotonic Dystrophy Type 1

[Molecular Therapy] Investigators presented an AAV-RNAi based strategy for dystrophia myotonica protein kinase (DMPK) reduction based on a muscle-targeted platform comprising an AAV capsid with high muscle transduction efficiency, a promoter with strong activity in muscle, and a DMPK-targeting artificial miRNA.

Cas9 Senses CRISPR RNA Abundance to Regulate CRISPR Spacer Acquisition

[Nature] The authors uncovered the first biological role for Cas9 that is independent of its dual RNA partners. Following depletion of crRNA and/or tracrRNA, Neisseria apoCas9 stimulates spacer acquisition efficiency.

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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