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Volume 8 Issue 2, February 2024

Stable transposon-driven integration of transgenes into immune cells

This issue highlights optimized base editors targeting the exon-7 mutation in SMN2 to restore the expression of the survival motor neuron protein to normal levels, the virus-mediated delivery of a transposon and an mRNA-encoded transposase for the integration of transgenes into immune cells, the enhanced viability of genome-engineered T cells transfected by electroporation via an isotonic buffer that dampens cytosolic cGAS–DNA interactions, the annotation of variants of the BRCA2 gene in human pluripotent stem cells, that host genes involved in viral processes can constrain the lentiviral delivery and expression of Cas13, and that replacing amino acid residues in an immunodominant and conserved T cell epitope in the capsid of an adeno-associated virus can abrogate its immunogenicity while preserving its function and potency.

The cover illustrates a gene-delivery system that enhances the stability of the integration of a desired transgene in immune cells by relying on a Sleeping Beauty transposase encoded into an mRNA delivered by an adeno-associated virus.

See Ye et al.

Image: Lupeng Ye and Sidi Chen. Cover design: Alex Wing.

Editorial

  • Appealing against an editor’s negative decision is likely to be more fruitful when considering the basis of the editorial assessment and offering ways forward.

    Editorial

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News & Views

  • Base editors can restore the expression of survival motor neuron protein to therapeutically beneficial levels in animal and cell models of spinal muscular atrophy.

    • Andrew Portell
    • Prashant Mali
    News & Views
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Research Briefings

  • We functionally assessed clinically observed mutations of the BRCA2 gene and analysed structure–function relationships of variants of the gene by using high-throughput CRISPR-mediated mutagenesis and pooled screening in locally haploid human pluripotent stem cells and in fibroblasts differentiated from them.

    Research Briefing
  • We revealed that the RNA-targeting activity of the Cas13 family of nucleases allows them to directly target endogenous RNA in mammalian cells. Such activity limits the usage of lentiviral Cas13 systems, and suggests a need for caution when applying Cas13-based systems.

    Research Briefing
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