Publications by authors named "Katerina Smetanova"
Large-scale manufacturing of base-edited chimeric antigen receptor T cells.
Mol Ther Methods Clin Dev
December 2023
Article Synopsis
- Base editing is a new gene-editing technique that allows targeted genetic changes without harmful DNA breaks, using enzymes usually delivered as costly linear mRNA.
- Researchers have developed a more affordable method using circular RNA (circRNA), which shows higher editing efficiency and reduces RNA requirements by eight times.
- This approach successfully produced a clinical dose of CAR T cells with up to 86% editing efficiency, highlighting circRNA's potential to lower costs and improve large-scale production of genetically modified cells.
The development of multicistronic vectors enabling differential transgene expression is a goal of gene therapy and poses a significant engineering challenge. Current approaches rely on the insertion of long regulatory sequences that occupy valuable space in vectors, which have a finite and limited packaging capacity. Here we describe a simple method of achieving differential transgene expression by inserting stop codons and translational readthrough motifs (TRMs) to suppress stop codon termination.
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