News & Views |
Featured
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Article
| Open Access
Improving prime editing with an endogenous small RNA-binding protein
Genome-scale genetic screens identify the small RNA-binding protein La as a strong mediator of prime editing.
- Jun Yan
- , Paul Oyler-Castrillo
- & Britt Adamson
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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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Article
| Open Access
Durable and efficient gene silencing in vivo by hit-and-run epigenome editing
Experiments in mice show that designed epigenome editors that contain domains of transcriptional repressors can enable stable epigenetic silencing of Pcsk9, a gene with a role in cholesterol homeostasis, without inducing DNA breaks.
- Martino Alfredo Cappelluti
- , Valeria Mollica Poeta
- & Angelo Lombardo
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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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Article
| Open Access
Epitope editing enables targeted immunotherapy of acute myeloid leukaemia
Epitope engineering of donor haematopoietic stem/progenitor cells endows haematopoietic lineages with selective resistance to CAR T cells or monoclonal antibodies, without affecting protein function or regulation, enabling the targeting of genes that are essential for leukaemia survival and reducing the risk of tumour immune escape.
- Gabriele Casirati
- , Andrea Cosentino
- & Pietro Genovese
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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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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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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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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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Article |
AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice
The feasibility of adeno-associated-virus-delivered nonsense suppressor tRNAs operating on premature termination codons (AAV-NoSTOP) is explored to restore gene function, using a mouse model of mucopolysaccharidosis type I for proof of concept.
- Jiaming Wang
- , Yue Zhang
- & Dan Wang
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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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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 |
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
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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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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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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
| Open Access
The NIH Somatic Cell Genome Editing program
This Perspective discusses how the Somatic Cell Genome Editing Consortium aims to accelerate the implementation of safe and effective genome-editing therapies in the clinic.
- Krishanu Saha
- , Erik J. Sontheimer
- & Jiangbing Zhou
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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 |
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
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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
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News |
‘CRISPR babies’ are still too risky, says influential panel
The safety and efficacy of genome editing in human embryos hasn’t been proven, researchers warn.
- Heidi Ledford
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Outlook |
Cystic fibrosis
Innovative therapies are bringing hope to people with this inherited lung disorder.
- Herb Brody
Targeting RNA opens therapeutic avenues for Timothy syndrome