Featured
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Outlook |
Cystic fibrosis
Innovative therapies are bringing hope to people with this inherited lung disorder.
- Herb Brody
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Outlook |
Gene therapy could offer an inclusive cure for cystic fibrosis
After three decades of false starts, gene therapy against the disease is in new clinical trials — and there is even hope of a cure.
- Roxanne Khamsi
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News & Views |
Mitochondrial genome editing gets precise
A bacterial toxin has been found that allows DNA in a cellular organelle called the mitochondrion to be precisely altered. This development could help to combat diseases caused by mutations in mitochondrial DNA.
- Magomet Aushev
- & Mary Herbert
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Article |
A bacterial cytidine deaminase toxin enables CRISPR-free mitochondrial base editing
An interbacterial toxin that catalyses the deamination of cytidines within double-stranded DNA forms part of a CRISPR-free, RNA-free base editing system that enables manipulation of human mitochondrial DNA.
- Beverly Y. Mok
- , Marcos H. de Moraes
- & David R. Liu
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Research Highlight |
Gene therapy shows promise for a disease that steals young men’s vision
Trial shows the safety of a treatment for X-linked retinitis pigmentosa.
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Innovations In |
Gene Therapy Arrives
After false starts, drugs that manipulate the code of life are finally changing lives.
- Jim Daley
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Innovations In |
All of Us
DNA-based medicine needs more diversity to avoid harmful bias. One big research project is fixing that.
- Stephanie Devaney
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Innovations In |
23 and Baby
We now have the ability to screen for thousands of genetic diseases in newborns. That may not always be the healthy thing to do.
- Tanya Lewis
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Innovations In |
The Power of Spheres
DNA or RNA molecules, arranged into spherical shapes, can attack brain cancers and other illnesses that evade conventional drug design.
- Chad A. Mirkin
- , Christine Laramy
- & Kacper Skakuj
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Editorial |
Expensive treatments for genetic disorders are arriving. But who should foot the bill?
The majority of people with sickle-cell disease are live in the world’s poorest communities and cannot afford the eye-watering costs of treatments.
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News Feature |
Gene therapy is facing its biggest challenge yet
After finally gaining traction as a potential treatment for certain genetic disorders, gene therapy tackles the challenge of sickle-cell disease.
- Heidi Ledford
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Nature Podcast |
Podcast: Quantum supremacy and ancient mammals
Listen to the latest from the world of science, with Nick Howe and Shamini Bundell.
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Article |
Search-and-replace genome editing without double-strand breaks or donor DNA
A new DNA-editing technique called prime editing offers improved versatility and efficiency with reduced byproducts compared with existing techniques, and shows potential for correcting disease-associated mutations.
- Andrew V. Anzalone
- , Peyton B. Randolph
- & David R. Liu
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News |
Russian ‘CRISPR-baby’ scientist has started editing genes in human eggs with goal of altering deaf gene
Denis Rebrikov also told Nature that he does not plan to implant gene-edited embryos until he gets regulatory approval.
- David Cyranoski
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Nature Index |
Stem-cell and genetic therapies make a healthy marriage
This scientific partnership could fight everything from blood diseases to HIV.
- Bianca Nogrady
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News |
Experimental gene therapy frees ‘bubble-boy’ babies from a life of isolation
Treatment restores immune-system function in young children with severe disorder.
- Heidi Ledford
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Outlook |
Four technologies that could transform the treatment of blindness
A decade ago, clinicians had nothing to offer most people affected by retinal degeneration. Breakthroughs in genetics, bionics and stem-cell therapy are changing that.
- Simon Makin
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Letter |
Precise therapeutic gene correction by a simple nuclease-induced double-stranded break
Disease-causing microduplications can be corrected by harnessing an endogenous double-stranded break DNA repair pathway.
- Sukanya Iyer
- , Sneha Suresh
- & Scot A. Wolfe
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Letter |
CAR T cell trogocytosis and cooperative killing regulate tumour antigen escape
Chimeric antigen receptors (CARs) promote antigen loss in tumour cells by trogocytosis, which results in T cell fratricide killing and exhaustion but can be counteracted by cooperative killing and combinatorial targeting.
- Mohamad Hamieh
- , Anton Dobrin
- & Michel Sadelain
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News |
World Health Organization panel weighs in on CRISPR-babies debate
Advisory committee calls for a global registry of studies that involve editing the human genome.
- Sara Reardon
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Article |
CasX enzymes comprise a distinct family of RNA-guided genome editors
CRISPR–CasX represents a distinct RNA-guided platform that is functionally separate from Cas9 and Cas12a and is active for bacterial and human genome modification.
- Jun-Jie Liu
- , Natalia Orlova
- & Jennifer A. Doudna
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Outlook |
Gene therapy
Gene tinkering opens the door to treatments for an array of diseases
- Herb Brody
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Outlook |
Gene therapy targets sickle-cell disease
The research is promising, but a true cure for this painful condition could be years away.
- Anna Nowogrodzki
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Outlook |
Gene therapy targets epilepsy
Anti-seizure medication doesn’t work in every person with epilepsy. But a treatment option is emerging that would spare the need for brain surgery.
- Liam Drew
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Outlook |
A genetically augmented future
Gene therapy could one day be used for bodily enhancement, creating an ethical minefield for physicians, says Ellen Wright Clayton.
- Ellen Wright Clayton
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Outlook |
Designer viruses could be the secret to cheaper and better gene therapy
Researcher and entrepreneur Luk Vandenberghe thinks he can transport genes into cells much more efficiently by improving the viral vectors that carry them.
- Neil Savage
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Outlook |
Change the genes to fix the skin
The largest organ in the body is a prime target for gene therapy.
- Kat Arney
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Outlook |
Prenatal gene therapy offers the earliest possible cure
Cancelling out an illness before a child is born offers the most potential benefit.
- Sarah DeWeerdt
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Outlook |
Access and affordability for all
The hope of gene therapy could be crushed by its financial burden unless there are more rational ways of paying for it, says Michael Sherman.
- Michael Sherman
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Outlook |
Regulating the gene-therapy revolution
The medical regulatory authorities ride a wave of clinical studies for gene therapies.
- Eric Bender
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News |
How the genome-edited babies revelation will affect research
Some scientists worry the startling claim will lead to knee-jerk regulations and damage the public’s trust in gene editing.
- David Cyranoski
- & Heidi Ledford
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News Feature |
How Facebook and Twitter could be the next disruptive force in clinical trials
Participants in medical research are more empowered than ever to influence the design and outcomes of experiments. Now, researchers are trying to keep up.
- Heidi Ledford
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Letter |
In vivo CRISPR editing with no detectable genome-wide off-target mutations
A strategy developed to define off-target effects of gene-editing nucleases in whole organisms is validated and leveraged to show that CRISPR–Cas9 nucleases can be used effectively in vivo without inducing detectable off-target mutations.
- Pinar Akcakaya
- , Maggie L. Bobbin
- & J. Keith Joung
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News |
First test of in-body gene editing shows promise
Preliminary results suggest that treatment for rare disease is safe, but its effectiveness is unclear.
- Heidi Ledford
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News |
Gene-silencing technology gets first drug approval after 20-year wait
The US Food and Drug Administration’s decision breathes new life into RNA-interference therapies.
- Heidi Ledford
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Nature Podcast |
Podcast: DNA scaffolds, climate-altering microbes, and a robot chemist
Listen to the latest science news, brought to you by Benjamin Thompson and Noah Baker.
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News |
Gene therapy in mouse fetuses treats deadly disease
The method could minimize damage from disease if a condition is diagnosed in utero.
- Heidi Ledford
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News |
CRISPR gene editing produces unwanted DNA deletions
DNA-cutting enzyme used for genetic modification can create large deletions and shuffle genes.
- Heidi Ledford
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Letter |
Reprogramming human T cell function and specificity with non-viral genome targeting
A non-viral strategy to introduce large DNA sequences into T cells enables the correction of a pathogenic mutation that causes autoimmunity, and the replacement of an endogenous T-cell receptor with an engineered receptor that can recognize cancer antigens.
- Theodore L. Roth
- , Cristina Puig-Saus
- & Alexander Marson
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Nature Podcast |
Backchat: Lab health, email briefings, and CRISPR
Benjamin Thompson hosts our regular roundtable discussion, with guests Heidi Ledford, Flora Graham, and Richard van Noorden.
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News |
Genetically modified bacteria enlisted in fight against disease
Engineered strains of E. coli and other microbes are being tested in people to combat a slew of illnesses.
- Sara Reardon
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Outlook |
How the gene behind Huntington’s disease could be neutralized
Antisense oligonucleotides are providing researchers and patients with fresh hope of targeting the condition’s genetic cause.
- Liam Drew
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Outlook |
CRISPR takes on Huntington’s disease
Gene editing offers the prospect of curing the inherited neurodegenerative condition in a single dose.
- Michael Eisenstein
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Editorial |
How to get public engagement right
Dialogue with the public requires a nuanced approach and a willingness to accept uncomfortable truths.
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Outlook |
A CRISPR edit for heart disease
A one-off injection to reduce the risk of cardiovascular disease is now a prospect thanks to advances in gene editing.
- Anthony King
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News |
Powerful enzyme could make CRISPR gene-editing more versatile
Revamped Cas9 protein could work on more sites in the genome, and with fewer unwanted effects.
- Heidi Ledford
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News Explainer |
How the immune system could stymie some CRISPR gene therapies
Researchers hoping to use a gene-editing technique to treat diseases may have to seek alternative enzymes.
- Heidi Ledford