Biotechnology articles from across Nature Portfolio

Lipid nanoparticle–mRNA complexes hold promise for treating cardiac diseases, but their transfection efficiency into the heart is limited. Using our previously developed human cardiac microphysiological screen, we identified modified lipid nanoparticle–mRNA complexes with improved transfection and gene editing efficacy, both in 3D micromuscles and in vivo in mice.

A synthetic peptide nanoparticle and microfluidic chip enable sterility testing of cell- and gene-based therapeutics in under 18 hours, bridging a long-standing safety gap for biopharmaceutical products that cannot undergo terminal sterilization and have limited shelf life.

We applied emerging label-free biophysical techniques to map the size, composition and shape of RNA lipid nanoparticles. By linking these physical measurements to gene expression in human T cells and mice, we uncovered structure–activity relationships that guide RNA delivery.

As tissue engineering has developed it has become apparent that multiple inputs are needed to fully replicate tissues, neuronal-axonal networks are an important part of this. In this review, the authors explore the advances in biomanufacturing and multi cell techniques aiming to generate tissues with integrated neuronal networks.

CRISPR base editing enables the precise introduction of single-nucleotide mutations in the genome. Here, authors generated new adenine/cytosine base editor dataset and proposed a deep-learning model CRISPRon-BE for base editor efficiency prediction, thereby enhancing base editing applications.

Synthetic biology is defined by Design-Build-Test-Learn cycles. Recent advances in machine learning are changing the landscape; thus, we propose that “Learning” can precede “Design”. Moreover, adopting cell-free platforms can further accelerate “Building” and “Testing” for megascale data generation and models.

Lipid nanoparticle–mRNA complexes hold promise for treating cardiac diseases, but their transfection efficiency into the heart is limited. Using our previously developed human cardiac microphysiological screen, we identified modified lipid nanoparticle–mRNA complexes with improved transfection and gene editing efficacy, both in 3D micromuscles and in vivo in mice.

In this Tools of the Trade article, Frank F. Lanfranchi describes Neuropixels 1.0 NHP, which allows the tracking of co-ordinated activity of hundreds of neurons across different brain areas in non-human primates, revealing the complexity of neural dynamics during complex tasks.

In a world first, a bespoke gene-editing therapy benefited one child. Now researchers plan to launch a clinical trial of the approach.

Concerns associated with the immunogenicity of mRNA lipid nanoparticles containing polyethylene glycol (PEG) are inspiring the development of PEG alternatives.