Collection 24 May 2022

Genomic medicine

Advances in genetic technologies and in the understanding of clinically relevant genetic variation are revolutionising how a whole range of diseases can be diagnosed and treated. This collection of articles offers a glimpse into research that is laying important groundwork for genomic medicine to play a transformative part in patient healthcare. The selected articles explore new approaches to prevent infectious disease, treat cancer and, potentially, cure inherited disorders using knowledge of the genome.

This Collection is editorially independent, produced with financial support from a third party. About this content.

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Regulated control of gene therapies by drug-induced splicing

A switch system is developed to control the expression of therapeutic genes, involving the administration of a small-molecule drug to induce splicing-mediated control of mRNA translation.
- Alex Mas Monteys
- Amiel A. Hundley
- Beverly L. Davidson
Article28 Jul 2021 Nature
Base editing of haematopoietic stem cells rescues sickle cell disease in mice

A custom adenine base editor can edit the variant of the β-globin gene that causes sickle cell disease into a non-pathogenic variant in human and mouse cells, and transplantation of the edited cells rescues sickle cell disease in mice.
- Gregory A. Newby
- Jonathan S. Yen
- David R. Liu
Article2 Jun 2021 Nature
Cas9 gene therapy for Angelman syndrome traps Ube3a-ATS long non-coding RNA

Genomic integration of an adeno-associated virus vector in a mouse model of Angelman syndrome unsilences paternal Ube3a and rescues anatomical and behavioural phenotypes, suggesting a pathway towards the treatment of this neurodevelopmental disorder.
- Justin M. Wolter
- Hanqian Mao
- Mark J. Zylka
Article21 Oct 2020 Nature
Personalized RNA mutanome vaccines mobilize poly-specific therapeutic immunity against cancer

The authors report the first-in-human application of personalized neo-antigen RNA vaccines in patients with melanoma.
- Ugur Sahin
- Evelyna Derhovanessian
- Özlem Türeci
Letter5 Jul 2017 Nature
Zika virus protection by a single low-dose nucleoside-modified mRNA vaccination

A single, low-dose intradermal immunization with lipid-nanoparticle-encapsulated nucleoside-modified mRNA encoding the pre-membrane and envelope glycoproteins of Zika virus protects both mice and rhesus macaques against infection and elicits rapid and long-lasting neutralizing antibody responses.
- Norbert Pardi
- Michael J. Hogan
- Drew Weissman
Letter2 Feb 2017 Nature
Delivering the next generation of breakthrough genomic medicines

Sponsor Feature24 May 2022

Genomic medicine

Content

Regulated control of gene therapies by drug-induced splicing

Base editing of haematopoietic stem cells rescues sickle cell disease in mice

Cas9 gene therapy for Angelman syndrome traps Ube3a-ATS long non-coding RNA

Personalized RNA mutanome vaccines mobilize poly-specific therapeutic immunity against cancer

Zika virus protection by a single low-dose nucleoside-modified mRNA vaccination

Delivering the next generation of breakthrough genomic medicines

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