ArticleMolecular therapy. Nucleic acids2023
Efficient shRNA-based knockdown of multiple target genes for cell therapy using a chimeric miRNA cluster platform.
Article in Molecular therapy. Nucleic acids, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers.
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Who cites it
13 citing papers in PubMed, 22 citations in OpenAlex.
- Adapter-based allogeneic CAR T cells to overcome antigen escape in solid tumors.Frontiers in immunology · 2026Review
- Cryopreservation of siRNA-Treated Cells Is Feasible.Nucleic acid therapeutics · 2025Article
- Article
- Analysis of Processing, Post-Maturation, and By-Products of shRNA in Gene and Cell Therapy Applications.Methods and protocols · 2025Article
- Clinical Proof-of-Concept of a Non-Gene Editing Technology Using miRNA-Based shRNA to Engineer Allogeneic CAR T-Cells.International journal of molecular sciences · 2025Article
- Universal CAR cell therapy: Challenges and expanding applications.Translational oncology · 2025Review
- Therapeutic Innovations in Nanomedicine: Exploring the Potential of Magnetotactic Bacteria and Bacterial Magnetosomes.International journal of nanomedicine · 2025Review
- Multiplex engineering using microRNA-mediated gene silencing in CAR T cells.Frontiers in immunology · 2025Article
- YTHDC1-mediated microRNA maturation is essential for hematopoietic stem cells maintenance.Cell death discovery · 2024Article
- Engineering immune-evasive allogeneic cellular immunotherapies.Nature reviews. Immunology · 2024Review
- Review
- Engineering strategies to safely drive CAR T-cells into the future.Frontiers in immunology · 2024Review
- Chimeric miRNA cluster enables multiplex and titratable gene inhibition in CAR-T cells.Molecular therapy. Nucleic acids · 2023Article
Corrections and comments
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Authors and funding
9 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Genome engineering technologies are powerful tools in cell-based immunotherapy to optimize or fine-tune cell functionalities. However, their use for multiple gene edits poses relevant biological and technical challenges. Short hairpin RNA (shRNA)-based cell engineering bypasses these criticalities and represents a valid alternative to CRISPR-based gene editing. Here, we describe a microRNA (miRNA)-based multiplex shRNA platform obtained by combining highly efficient miRNA scaffolds into a chimeric cluster, to deliver up to four shRNA-like sequences. Thanks to its limited size, our cassette could be deployed in a one-step process along with all the CAR components, streamlining the generation of engineered CAR T cells. The plug-and-play design of the shRNA platform allowed us to swap each shRNA-derived guide sequence without affecting the system performance. Appropriately choosing the target sequences, we were able to either achieve a functional KO, or fine-tune the expression levels of the target genes, all without the need for gene editing. Through our strategy we achieved easy, safe, efficient, and tunable modulation of multiple target genes simultaneously. This approach allows for the effective introduction of multiple functionally relevant tweaks in the transcriptome of the engineered cells, which may lead to increased performance in challenging environments, e.g., solid tumors.
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.