Featured
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Article |
Towards germline gene therapy of inherited mitochondrial diseases
Mutations in mitochondrial DNA cause a wide range of disorders in humans, with a high prevalence; here it is shown that the nucleus of an affected woman’s egg could be inserted into healthy donor egg cytoplasm by spindle transfer, allowing the birth of healthy offspring.
- Masahito Tachibana
- , Paula Amato
- & Shoukhrat Mitalipov
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World View |
Gene therapies need new development models
As with other medicines, the approval of gene therapies should hinge on a risk–benefit analysis for the patient, argues Fulvio Mavilio.
- Fulvio Mavilio
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News |
Gene therapy restores sense of smell to mice
Cilia repair rescues olfactory function, but its potential in broader disorders is unclear.
- Melissa Lee Phillips
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Research Highlights |
Gene fix repairs hearing
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News |
Europe nears first approval for gene therapy
Treatment for rare fat-processing disease gains approval from medicines regulator.
- Daniel Cressey
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Research Highlights |
Quit smoking with a shot
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Research Highlights |
An eye for gene repair
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News |
French institute prepares for gene-therapy push
Genethon relaunches itself as a force for translational medicine.
- Alison Abbott
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News |
UK sets sights on gene therapy in eggs
Public consultation and safety assessment would pave the way for embryo manipulation to treat genetic diseases.
- Ewen Callaway
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Seven Days |
Seven days: 9–15 December 2011
The week in science: boost for gene therapy; EPA reports concern over fracking; and a fresh clue to ancient water on Mars.
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News |
Gene therapy can protect against HIV
An introduced gene conveys long-lived resistance to HIV infection in mice.
- Lauren Gravitz
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News |
Liver-disease mutation corrected in human stem cells
Patient-derived stem cells reprogrammed to become healthier.
- Susan Young
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Research Highlights |
Long-term fix for SCID kids
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News |
Gene-therapy successes spur hope for embattled field
Technique now being tested in a range of conditions.
- Erika Check Hayden
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News |
Gene-therapy enzymes make unpredicted errors
Techniques show mistakes of 'highly specific' molecular tools.
- Heidi Ledford
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Research Highlights |
Gene repair tool for stem cells
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News |
Gene therapy offers hope for Parkinson's disease
Small trial improves patients' symptoms.
- Ewen Callaway
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News |
Targeted gene editing enters clinic
Patients with HIV first to receive experimental gene therapy.
- Heidi Ledford
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News & Views |
A molecular memory booster
For many – if not all – of us, cognitive enhancement is desirable, but agents that would truly improve memory are hard to find. Unexpectedly, the product of an imprinted gene emerges as a promising candidate. See Article p.491
- Johannes Gräff
- & Li-Huei Tsai
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Research Highlights |
Gene therapy: Small RNAs aid cell transplants
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News |
Gene therapy helps depressed mice
Revving up expression of a single gene in the brain reverses depression symptoms
- Alla Katsnelson
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Letter |
Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia
Disorders caused by abnormal β-globin, such as β-thalassaemia, are the most prevalent inherited disorders worldwide. For treatment, many patients are dependent on blood transfusions; thus far the only cure has involved matched transplantation of haematopoietic stem cells. Here it is shown that lentiviral β-globin gene transfer can be an effective substitute for regular transfusions in a patient with severe β-thalassaemia.
- Marina Cavazzana-Calvo
- , Emmanuel Payen
- & Philippe Leboulch
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Books & Arts |
Secrets of a long life
Two books on ageing understate the challenges of prolonging a healthy lifespan, finds Caleb Finch.
- Caleb Finch
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News & Views |
Targeting β-thalassaemia
Patients with disorders of the blood protein haemoglobin often depend on lifelong blood transfusions. That could change, given the success of gene therapy in a patient with one such disorder.
- Derek A. Persons
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News |
Gene-therapy hope for β-thalassaemia patients
A defective haemoglobin gene has been successfully replaced with a healthy copy.
- Joseph Milton
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News Feature |
Biotechnology: Crossing the barrier
Researchers have rallied round a promising molecule for rescuing dying nerves. But getting it into the brain remains a daunting challenge, finds Brian Vastag.
- Brian Vastag
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News |
Triple-punch gene therapy targets HIV
Stem-cell transplant passes safety trial.
- Alla Katsnelson
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News |
Freeing human eggs of mutant mitochondria
Transmission of mitochondrial diseases from mother to offspring could be prevented.
- Alla Katsnelson
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Letter |
Hsp70 stabilizes lysosomes and reverts Niemann–Pick disease-associated lysosomal pathology
Heat shock protein 70 (Hsp70) is a molecular chaperone which, by inhibiting lysosomal membrane permeabilization, promotes the survival of stressed cells. Hsp70 is now shown to stabilize lysosomes by binding to an anionic phospholipid, BMP, resulting in stimulation of acid sphingomyelinase (ASM) activity. Notably, the decreased ASM activity and lysosomal stability seen in patients with Niemann–Pick disease can be corrected by treatment with recombinant Hsp70.
- Thomas Kirkegaard
- , Anke G. Roth
- & Marja Jäättelä