Featured
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Research Highlight |
A CRISPR system to turn genes on
New technique modifies gene expression instead of actual DNA.
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News & Views |
Transgenic stem cells replace skin
The treatment of a patient affected by an incurable genetic skin disease demonstrates the efficacy, feasibility and safety of replacing almost the whole skin using genetically corrected stem cells. See Letter p.327
- Mariaceleste Aragona
- & Cédric Blanpain
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Article |
Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage
A new DNA ‘base editor’ can change targeted A•T base pairs to G•C, allowing disease-associated mutations to be corrected and disease-suppressing mutations to be introduced into cells.
- Nicole M. Gaudelli
- , Alexis C. Komor
- & David R. Liu
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News |
FDA advisers back gene therapy for rare form of blindness
Therapy that targets disease-causing mutations could become the first of its kind approved for use in the United States.
- Heidi Ledford
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Outlook |
Gene therapy: Erasing sickle-cell disease
Clinical trials may soon test whether gene editing can cure a group of debilitating haemoglobin disorders.
- Katherine Bourzac
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Letter |
Enhanced proofreading governs CRISPR–Cas9 targeting accuracy
A new engineered version of SpCas9, called HypaCas9, displays enhanced accuracy of editing without significant loss of efficiency at the desired target.
- Janice S. Chen
- , Yavuz S. Dagdas
- & Jennifer A. Doudna
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News |
Doubts raised about CRISPR gene-editing study in human embryos
Alternative explanations challenge whether technique actually fixed a genetic mutation as claimed.
- Ewen Callaway
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News |
CRISPR fixes disease gene in viable human embryos
Gene-editing experiment pushes scientific and ethical boundaries.
- Heidi Ledford
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Letter |
Targeting a CAR to the TRAC locus with CRISPR/Cas9 enhances tumour rejection
Introducing chimeric antigen receptors into the endogenous T-cell receptor locus reduces tonic signalling, averts accelerated T-cell differentiation and delays T-cell exhaustion, leading to enhanced function and anti-tumour efficacy compared to random integrations.
- Justin Eyquem
- , Jorge Mansilla-Soto
- & Michel Sadelain
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News |
US science advisers outline path to genetically modified babies
Modified human embryos should be allowed if researchers meet strict criteria, says long-awaited National Academies report.
- Sara Reardon
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Research Highlights |
Gene therapy restores hearing
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News |
CRISPR gene-editing tested in a person for the first time
The move by Chinese scientists could spark a biomedical duel between China and the United States.
- David Cyranoski
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News |
Safety concerns blight promising cancer therapy
As the first T-cell therapies for tumours near US approval, researchers race to engineer less-toxic alternatives.
- Heidi Ledford
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News |
CRISPR deployed to combat sickle-cell anaemia
Studies in mice highlight the promises — and challenges — of CRISPR–Cas9 gene editing.
- Heidi Ledford
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News |
UK bioethicists eye designer babies and CRISPR cows
Panel will focus on the implications of gene editing in human reproduction and livestock.
- Heidi Ledford
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Outlook |
Lysosomal storage disorders: 4 big questions
The quest to understand lysosomal storage disorders (LSDs) has left researchers grappling with questions that have implications for other diseases too.
- Michael Eisenstein
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Outlook |
Gene therapy: A new chapter
In the 1980s, bone-marrow transplants transformed the lives of children with fatal lysosomal diseases. Researchers are now working on another revolutionary treatment.
- Anthony King
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Outlook |
Gene therapy: Industrial strength
After a series of setbacks, genetic therapies are finally moving beyond small academic trials towards approval as treatments.
- Eric Bender
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News |
Replications, ridicule and a recluse: the controversy over NgAgo gene-editing intensifies
As failures to replicate results using the CRISPR alternative stack up, a quiet scientist stands by his claims.
- David Cyranoski
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News |
Chinese scientists to pioneer first human CRISPR trial
Gene-editing technique to treat lung cancer is due to be tested in people in August.
- David Cyranoski
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Editorial |
Gene-therapy trials must proceed with caution
The perils of the past must not be allowed to happen again
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News |
First CRISPR clinical trial gets green light from US panel
The technique's first test in people could begin as early as the end of the year.
- Sara Reardon
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News |
Promising gene therapies pose million-dollar conundrum
Economists, investors and medical insurers can’t figure out how to pay for cutting-edge drugs.
- Erika Check Hayden
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Letter |
Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage
CRISPR/Cas9 DNA editing creates a double-stranded break in the target DNA, which can frequently generate random insertion or deletion of bases (indels); a new genome editing approach combining Cas9 with a cytidine deaminase is described here, which corrects point mutations more efficiently than canonical Cas9, while avoiding double-stranded breaks and indel formation.
- Alexis C. Komor
- , Yongjoo B. Kim
- & David R. Liu
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News |
CRISPR: Ask us anything
Join us on Reddit where we'll be talking about all things CRISPR.
- Heidi Ledford
- & Sara Reardon
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News & Views |
The domestication of Cas9
The enzyme Cas9 is used in genome editing to cut selected DNA sequences, but it also creates breaks at off-target sites. Protein engineering has now been used to make Cas9 enzymes that have minimal off-target effects. See Article p.490
- Fyodor Urnov
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Letter |
An essential receptor for adeno-associated virus infection
An adeno-associated virus (AAV) receptor protein essential for AAV2 entry into cells is identified; AAV receptor binds directly to the virus, and its ablation renders a diverse range of mammalian cell types and mice resistant to infection by AAV of multiple serotypes.
- S. Pillay
- , N. L. Meyer
- & J. E. Carette
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Research Highlights |
CRISPR fixes muscle disease
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Article |
High-fidelity CRISPR–Cas9 nucleases with no detectable genome-wide off-target effects
A high-fidelity variant of Streptococcus pyogenes CRISPR–Cas9 is reported that lacks detectable off-target events as assessed by genome-wide break capture and targeted sequencing methods.
- Benjamin P. Kleinstiver
- , Vikram Pattanayak
- & J. Keith Joung
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News |
Gene-editing summit supports some research in human embryos
Three-day meeting calls for further discussions on modifications to the gene pool.
- Sara Reardon
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Outlook |
Research: Biology's big hit
Scientists now have several tools to edit the genomes of living organisms. One of the most recent is revolutionizing research and has thrust two of its creators into the limelight.
- Zoë Corbyn
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News |
Human-genome editing summit to sample global attitudes
Academies in the US, China and the UK jointly organized the gathering.
- Sara Reardon
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News |
Leukaemia success heralds wave of gene-editing therapies
One-year-old girl treated as plans to inject DNA-cutting technology directly into patients' bodies take shape.
- Sara Reardon
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News |
Success against blindness encourages gene therapy researchers
Positive news buoys a beleaguered field, but treatment benefits may fade.
- Heidi Ledford
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News |
Gene-editing record smashed in pigs
Researchers modify more than 60 genes in effort to enable organ transplants into humans.
- Sara Reardon
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Research Highlights |
Ancestral virus for gene therapy
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Research Highlights |
Deafness can be reversed in mice
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Inside View |
Inside View: Glaxosmithkline
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Research Highlights |
Gene therapy halts type 1 diabetes
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News |
Chinese scientists genetically modify human embryos
Rumours of germline modification prove true — and look set to reignite an ethical debate.
- David Cyranoski
- & Sara Reardon
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News |
Mini enzyme moves gene editing closer to the clinic
Discovery expands potential CRISPR toolbox for treating genetic diseases in humans.
- Heidi Ledford
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Article |
In vivo genome editing using Staphylococcus aureus Cas9
The physical size of the commonly used Cas9 from Streptococcus pyogenes poses challenges for CRISPR-Cas genome editing systems that use the adeno-associated virus as a delivery vehicle; here, smaller Cas9 orthologues are characterized, and Cas9 from Staphylococcus aureus allowed targeting of the cholesterol regulatory gene Pcsk9 in the mouse liver.
- F. Ann Ran
- , Le Cong
- & Feng Zhang
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News & Views |
Tied down by its own receptor
An engineered protein that binds to the envelope of HIV viruses protects monkeys against infection with a simian–human virus that causes AIDS. This gene-therapy approach might provide an alternative to elusive HIV vaccines. See Letter p.87
- Nancy L. Haigwood
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Letter |
Promoterless gene targeting without nucleases ameliorates haemophilia B in mice
Promoterless recombinant adeno-associated virus is used without nucleases to target the human coagulation factor IX gene to the liver-expressed albumin locus in haemophilia B mice, with an on-target integration into ∼0.5% of the albumin alleles in hepatocytes; stable F9 plasma levels at 7–20% of normal were obtained, leading to normal coagulation times in treated factor-IX-deficient mice.
- A. Barzel
- , N. K. Paulk
- & M. A. Kay
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Article |
Targeted genome editing in human repopulating haematopoietic stem cells
The feasibility of targeted genome editing in human haematopoietic stem cells is demonstrated; the study overcomes previously existing barriers by tailoring delivery platforms and culture conditions.
- Pietro Genovese
- , Giulia Schiroli
- & Luigi Naldini
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Letter |
Cell-autonomous correction of ring chromosomes in human induced pluripotent stem cells
Generation of human induced pluripotent stem cells from patient fibroblasts containing ring chromosomes with large deletions reveals that reprogrammed cells lose the abnormal chromosome and duplicate the wild-type homologue through compensatory uniparental disomy, suggesting that cellular reprogramming may hold potential for ‘chromosome therapy’.
- Marina Bershteyn
- , Yohei Hayashi
- & Anthony Wynshaw-Boris
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Letter |
Selection and evaluation of clinically relevant AAV variants in a xenograft liver model
Chimaeric human–murine adeno-associated virus (AAV) capsids are described that transduce human primary hepatocytes more efficiently than currently used AAV vectors; the novel vectors may be good clinical candidates.
- Leszek Lisowski
- , Allison P. Dane
- & Mark A. Kay
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Article |
Translating dosage compensation to trisomy 21
This study uses zinc-finger nucleases to target an inducible XIST transgene into chromosome 21 from trisomic Down’s syndrome pluripotent stem cells; the XIST RNA coats one copy of chromosome 21 and triggers whole chromosome silencing, suggesting the potential of this approach for studying chromosomal disorders such as Down’s syndrome and for research into gene therapies.
- Jun Jiang
- , Yuanchun Jing
- & Jeanne B. Lawrence