News & Views |
Featured
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Spotlight |
Stealthy stem cells to treat disease
Gene-editing strategies that allow stem cells to evade the immune system offer hope for universal cell-replacement therapies.
- Elie Dolgin
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News |
‘Epigenetic’ editing cuts cholesterol in mice
Changes to chemical tags on DNA in mice dial down the activity of a gene without cuts to the genome.
- Heidi Ledford
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Technology Feature |
Seven technologies to watch in 2024
Advances in artificial intelligence are at the heart of many of this year’s most exciting areas of technological innovation
- Michael Eisenstein
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News |
How CRISPR gene editing could help treat Alzheimer’s
Some researchers hoping that gene-editing technology can conquer forms of Alzheimer’s caused by genetic mutations.
- Tosin Thompson
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News |
CRISPR 2.0: a new wave of gene editors heads for clinical trials
Landmark approval of the first CRISPR therapy paves the way for treatments based on more efficient and more precise genome editors.
- Heidi Ledford
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News |
UK first to approve CRISPR treatment for diseases: what you need to know
The landmark decision could transform the treatment of sickle-cell disease and β-thalassaemia — but the technology is expensive.
- Carissa Wong
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News |
First trial of ‘base editing’ in humans lowers cholesterol — but raises safety concerns
Super-precise gene-editing approach switches off a gene in the liver that regulates ‘bad’ cholesterol.
- Miryam Naddaf
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News Explainer |
Is CRISPR safe? Genome editing gets its first FDA scrutiny
Advisers to the US regulatory agency will examine the safety profile of a CRISPR-based treatment for sickle-cell disease.
- Heidi Ledford
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News |
Gene therapies for rare diseases are under threat. Scientists hope to save them
As industry steps aside, scientists seek innovative ways to make sure expensive treatments can reach people who need them.
- Heidi Ledford
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News Feature |
‘It’s a vote for hope’: first gene therapy for muscular dystrophy nears approval, but will it work?
The FDA’s decision, expected this month, follows several setbacks and delays and will pose difficult choices for the families of children with Duchenne muscular dystrophy.
- Sara Reardon
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Editorial |
The gene-therapy revolution risks stalling if we don’t talk about drug pricing
Regulation and new intellectual property laws are needed to reduce the cost of gene-editing treatments and fulfil their promise to improve human health.
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News |
‘Astonishing’ molecular syringe ferries proteins into human cells
Technique borrowed from nature, and honed using artificial intelligence, could spur the development of better drug-delivery systems.
- Heidi Ledford
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Nature Index |
How Japanese science is trying to reassert its research strength
Successes in life sciences and international collaboration could be key to boosting the country’s high-quality output.
- Tim Hornyak
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Article
| Open Access
Aberrant phase separation and nucleolar dysfunction in rare genetic diseases
Frameshift mutations that create arginine-rich basic tails in transcription factors and other proteins can lead to altered phase separation in the nucleolus, which in turn leads to syndromes such as brachyphalangy, polydactyly and tibial aplasia.
- Martin A. Mensah
- , Henri Niskanen
- & Denes Hnisz
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News Feature |
The race to supercharge cancer-fighting T cells
With a slew of tools to trick out immune cells, researchers are expanding the repertoire of CAR-T therapies.
- Heidi Ledford
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Article
| Open Access
Mono- and biallelic variant effects on disease at biobank scale
An analysis of biobank data from the FinnGen project examines dosage effects of genetic variants on disease, andidentifies a benefit when considering more complex inheritance in the genetics of common as well as Mendelian diseases.
- H. O. Heyne
- , J. Karjalainen
- & M. J. Daly
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Nature Index |
Biomedical breakthroughs come of age
Approaches to therapy that have long been stuck in the lab are finally finding their way into the clinic.
- Benjamin Plackett
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Nature Index |
How gene therapy is emerging from its ‘dark age’
After years of setbacks, the field is starting to deliver on its promises.
- Gemma Conroy
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News |
Researchers welcome $3.5-million haemophilia gene therapy — but questions remain
The world’s most expensive drug has the potential to save lives. But it cannot treat the most common form of the disease.
- Miryam Naddaf
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Article
| Open Access
Nuclear-embedded mitochondrial DNA sequences in 66,083 human genomes
A study examining DNA transfer from mitochondria to the nucleus using whole-genome sequences from 66,083 people shows that this is an ongoing dynamic process in normal cells with distinct roles in different types of cancer.
- Wei Wei
- , Katherine R. Schon
- & Patrick F. Chinnery
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News |
CRISPR ‘cousin’ put to the test in landmark heart-disease trial
Gene-therapy test launches pivotal year for precise genome-editing technique known as base editing.
- Heidi Ledford
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News |
Gene therapy’s comeback: how scientists are trying to make it safer
Unwanted immune responses threaten to derail some gene therapies. But researchers are seeking ways to combat harmful inflammation.
- Heidi Ledford
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Analysis
| Open Access
A joint NCBI and EMBL-EBI transcript set for clinical genomics and research
Matched Annotation from NCBI and EMBL-EBI (MANE) delivers joint transcript sets from Ensembl/GENCODE and RefSeq for standardizing variant reporting in clinical genomics and research.
- Joannella Morales
- , Shashikant Pujar
- & Terence D. Murphy
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Review Article |
Human genetic and immunological determinants of critical COVID-19 pneumonia
The COVID Human Genetic Effort examines the molecular, cellular and immunological determinants of the various SARS-CoV-2-related disease manifestations by searching for causal errors of immunity.
- Qian Zhang
- , Paul Bastard
- & Jean-Laurent Casanova
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Technology Feature |
Seven technologies to watch in 2022
Our fifth annual round-up of the tools that look set to shake up science this year.
- Michael Eisenstein
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Article |
Disease variant prediction with deep generative models of evolutionary data
A new computational method, EVE, classifies human genetic variants in disease genes using deep generative models trained solely on evolutionary sequences.
- Jonathan Frazer
- , Pascal Notin
- & Debora S. Marks
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Innovations In |
Gene Therapy Is Coming of Age
Various approaches are approved for treating blood cancers and a few rare disorders—they may soon become standard care.
- Lauren Gravitz
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Innovations In |
The Definition of Gene Therapy Has Changed
Over the past few years the discipline has evolved in significant ways.
- Esther Landhuis
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Innovations In |
The Quest to Overcome Gene Therapy’s Failures
Tragic side effects plagued the field’s early years, but researchers are finding ways to minimize the risks.
- Tanya Lewis
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Innovations In |
Four Success Stories in Gene Therapy
The field is beginning to fulfill its potential. These therapies offer a glimpse of what’s to come.
- Jim Daley
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Innovations In |
We Need to Ground Truth Assumptions about Gene Therapy
Researchers, practitioners and patients must balance the discipline's promise with its reality.
- Marla Broadfoot
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Spotlight |
The gene-editing engineer working to boost expertise in China
The chief executive of Qihan Biotech aims to enable the first successful pig-to-human organ transplant and foster innovative biomedical research in her home country.
- Sarah O’Meara
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Outlook |
Sickle-cell disease
A condition that affects many people of African descent is finally meeting its therapeutic match.
- Herb Brody
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Outlook |
Can CRISPR cure sickle-cell disease?
Early trials show promise, but the challenge will be reaching those most in need.
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Outlook |
Gene therapies close in on a cure for sickle-cell disease
As multiple genetic strategies advance through the clinic, important safety questions remain to be answered.
- Michael Eisenstein
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Outlook |
Collect more data from Africa to improve gene therapy
Despite the continent being the epicentre of sickle-cell disease, too few Africans are included in genome-wide association studies.
- Ambroise Wonkam
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Article |
Deficient H2A.Z deposition is associated with genesis of uterine leiomyoma
Analyses of samples from 728 women with uterine leiomyomas (uterine fibroids), and public data, show that somatic and germline mutations in the SRCAP histone-loading complex genes are associated with the condition.
- Davide G. Berta
- , Heli Kuisma
- & Lauri A. Aaltonen
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News |
WHO should lead on genome-editing policy, advisers say
World Health Organization committee says it’s too soon to allow heritable gene editing, but points to paths forward for other applications.
- Heidi Ledford
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News Round-Up |
CRISPR-therapy promise, malaria vaccine and pandemic parenting
The latest science news, in brief.
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News |
Landmark CRISPR trial shows promise against deadly disease
Administering gene-editing treatment directly into the body could be a safe and effective way to treat a rare, life-threatening condition.
- Heidi Ledford
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Research Highlight |
Kids with ‘bubble baby disease’ see gains after gene therapy
Stem cells carrying corrective genes offer hope for a devastating immune disorder.
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News |
Failure of genetic therapies for Huntington’s devastates community
Hopes were high for drugs designed to lower levels of a mutant protein, but development has stalled.
- Diana Kwon
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Perspective |
Improving reporting standards for polygenic scores in risk prediction studies
An updated set of reporting standards for the development, interpretation and evaluation of polygenic risk scores is presented, which should aid the translation of these scores into clinical applications.
- Hannah Wand
- , Samuel A. Lambert
- & Genevieve L. Wojcik
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Article |
Non-coding deletions identify Maenli lncRNA as a limb-specific En1 regulator
The long non-coding RNA locus Maenli controls mouse limb development by regulating En1 activity, and the absence of the homolgous MAENLI locus is associated with severe congenital limb defects in humans.
- Lila Allou
- , Sara Balzano
- & Andrea Superti-Furga
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News & Views |
Base editor repairs mutation found in the premature-ageing syndrome progeria
No cure exists for the lethal premature-ageing condition Hutchinson–Gilford progeria. A gene-editing tool — adenine base editors — offers a way to treat the condition in mice. Could this approach lead to an effective therapy?
- Wilbert P. Vermeij
- & Jan H. J. Hoeijmakers
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Article |
In vivo base editing rescues Hutchinson–Gilford progeria syndrome in mice
In a mouse model of progeria, an adenine base editor delivered with adeno-associated virus corrects the pathogenic mutation in LMNA, rescues vascular pathology and markedly extends the lifespan of the mice.
- Luke W. Koblan
- , Michael R. Erdos
- & David R. Liu
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News |
CRISPR gene therapy shows promise against blood diseases
Researchers report early successes using genetic approaches to treat sickle-cell anaemia and β-thalassaemia.
- Heidi Ledford
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Article |
Evidence for 28 genetic disorders discovered by combining healthcare and research data
By integrating healthcare and exome-sequencing data from parent–offspring trios of patients with developmental disorders, 28 genes that had not previously been associated with developmental disorders were identified.
- Joanna Kaplanis
- , Kaitlin E. Samocha
- & Kyle Retterer
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Technology Feature |
A menagerie of stem-cell models
When conventional laboratory models fail, stem cells from squirrels, seals and other species can come to researchers’ aid.
- Jyoti Madhusoodanan
Targeting RNA opens therapeutic avenues for Timothy syndrome