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Despite their immense potential, gene and cell therapies that target rare diseases are at risk of market withdrawal, owing to several challenges. The authors describe these hurdles and call for innovative measures to improve the economic sustainability of gene and cell therapies after regulatory approval.
In this Tools of the Trade article, Nadiya Khyzha describes SLAM-RT&Tag, a method for profiling RNA localization and dynamics within nuclear compartments, such as speckles.
In this Tools of the Trade article, Timothy Olsen introduces DEFND-seq, a scalable method for co-sequencing RNA and DNA in single cells using commercially available high-throughput kits.
In this Journal Club, Kelley Harris recalls a 2003 paper by Goriely et al. that identified interesting evolutionary forces acting on mutations that are beneficial for cells within our bodies but detrimental to the health of our children.
In this Journal Club, Tracy Bedrosian describes a 2001 paper by Rehen et al. that provided early evidence of pervasive somatic variation throughout the human brain.
Collins and Talkowski provide a broad overview of structural variation in the human genome that covers their mutational properties, the dynamics of population genetics and functional consequences in disease as well as promising directions for future research.
In this Review, Dowdle and Lykke-Andersen discuss advances in our understanding of the machinery and mechanisms involved in the control of cytoplasmic mRNA decay and comment on implications for the design of therapeutic mRNAs.
In this Review, Isomura and Kageyama discuss how advances in live imaging, stem cell technologies and synthetic approaches are providing insights into the mechanisms underlying synchronization and species-specific periodicity of the mammalian segmentation clock during somitogenesis.
This Review explores how experimental models of metastasis, such as mouse models and cell cultures, can complement the (multi)omics analysis of human metastasis samples, thereby filling knowledge gaps left by model studies and validating the findings from human sequencing data.
