Featured
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Article |
BNT162b2 vaccine induces neutralizing antibodies and poly-specific T cells in humans
In a phase-I/II trial in healthy adults, the BNT162b2 vaccine induces neutralizing antibodies and poly-specific T cells against SARS-CoV-2 epitopes that are conserved in a wide range of currently circulating variants.
- Ugur Sahin
- , Alexander Muik
- & Özlem Türeci
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Article |
In vivo CRISPR base editing of PCSK9 durably lowers cholesterol in primates
In a cynomolgus macaque model, CRISPR base editors delivered in lipid nanoparticles are shown to efficiently and stably knock down PCSK9 in the liver to reduce levels of PCSK9 and low-density lipoprotein cholesterol in the blood.
- Kiran Musunuru
- , Alexandra C. Chadwick
- & Sekar Kathiresan
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Letter |
Biotechnological mass production of DNA origami
All necessary strands for DNA origami can be created in a single scalable process by using bacteriophages to generate single-stranded precursor DNA containing the target sequences interleaved with self-excising DNA enzymes.
- Florian Praetorius
- , Benjamin Kick
- & Hendrik Dietz
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Letter |
RNA targeting with CRISPR–Cas13
The class 2 type VI RNA-guided RNA-targeting CRISPR–Cas effector Cas13 can be engineered for RNA knockdown and binding, expanding the CRISPR toolset with a flexible platform for studying RNA in mammalian cells and therapeutic development.
- Omar O. Abudayyeh
- , Jonathan S. Gootenberg
- & Feng Zhang
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Letter |
MicroRNA silencing for cancer therapy targeted to the tumour microenvironment
A novel anti-microRNA delivery platform that targets the acidic tumour microenvironment, in which a chosen anti-miRNA is coupled to a peptide that can transport the anti-miRNA across cell membranes specifically in an acidic environment.
- Christopher J. Cheng
- , Raman Bahal
- & Frank J. Slack
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Article |
Functional screening identifies miRNAs inducing cardiac regeneration
The human heart regenerates poorly, causing insufficient healing after injury; here, microRNAs screened for the ability to induce cardiomyocyte proliferation are shown to stimulate cardiac regeneration and almost complete recovery of the heart after infarction.
- Ana Eulalio
- , Miguel Mano
- & Mauro Giacca
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News & Views |
Kill the messenger where it lives
A mutant repeating DNA sequence produces a toxic RNA molecule that causes the neuromuscular disorder myotonic dystrophy type 1. An ‘antisense’ therapy that targets this RNA in cell nuclei shows promise in mice. See Letter p.111
- Peter K. Todd
- & Henry L. Paulson
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Letter |
Targeting nuclear RNA for in vivo correction of myotonic dystrophy
Nuclear-retained transcripts containing expanded repeats are shown to be sensitive to antisense silencing, and in a transgenic mouse model of myotonic dystrophy type 1, systemic administration of ASOs causes a rapid knockdown of the toxic RNA in skeletal muscle, correcting some hallmark features of the disease.
- Thurman M. Wheeler
- , Andrew J. Leger
- & Charles A. Thornton
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Research Highlights |
Gene blocks effects of stress
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Research Highlights |
Silencing Huntington's gene
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Letter |
Inhibition of miR-33a/b in non-human primates raises plasma HDL and lowers VLDL triglycerides
- Katey J. Rayner
- , Christine C. Esau
- & Kathryn J. Moore
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Letter |
Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model
- Yimin Hua
- , Kentaro Sahashi
- & Adrian R. Krainer
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Letter |
Pathogenic exon-trapping by SVA retrotransposon and rescue in Fukuyama muscular dystrophy
- Mariko Taniguchi-Ikeda
- , Kazuhiro Kobayashi
- & Tatsushi Toda
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News |
Drug giants turn their backs on RNA interference
A once much-touted technique faces a difficult transition to the clinic.
- Heidi Ledford
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Research Highlights |
Drug development: Virus knockdown
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Technology Feature |
From tools to therapies
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Technology Feature |
Homing in on delivery
The scientific community now seems convinced that small RNAs will become therapies, if new tools can help these large molecules to make it safely into cells. Monya Baker reports.
- Monya Baker
Targeting Xist with compounds that disrupt RNA structure and X inactivation