Using 'base editing', researchers have cured progeria in mice. This genetic syndrome causes premature ageing in humans – those with the disease usually don't live past the age of 13.
Introducing healthy genes to replace defective ones is the essence of gene therapy.
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The immune system is trained to destroy viruses, even when they carry therapeutic cargo as is the case in gene therapy. Now researchers have figured out how to dial down the immune response.
Researchers are now testing treatments for several kinds of visual impairment.
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Genome sequencing technologies have transformed biological research in many ways, but have had a much smaller effect on the treatment of common diseases.
More than 1 million people in the U.S. are addicted to cocaine.
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Addiction to cocaine is wildly difficult to conquer. But physicians may soon have a new type of gene therapy for patients that makes the drug less alluring.
Delivering DNA to immune cells is the trickiest part of developing new gene-based therapies.
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Researchers are trying to boost the power of our immune system by genetically altering our white blood cells and transforming them into super-soldiers to fight cancer.
Gene therapy trials may mean that the next generation of children born with inherited eye diseases have treatment options.
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As we enter an era where once incurable diseases become curable, be prepared for some challenging debates about how to pay for gene therapy and the value of a human life.
Gene editing a fertilized human embryo.
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Scientists worldwide are calling for a moratorium on gene editing in germline cells. But what is a germline cell? How does it differ from other cells in our body? Why does it matter if we edit them?
Eugenics was previously the realm of social biology.
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If those who survive are the fittest, does that also make them the best? And if so, is engineering 'better' babies just evolution, or another step in a long history of eugenics?
Determining the structure of the DNA was the beginning of the gene therapy journey.
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Once genetic lesions for diseases such as cystic fibrosis and haemophilia were identified, the idea of replacing or correcting defective genes grew into what we now call "gene therapy".
Lines of cocaine.
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In a completely new approach to treating addiction, researchers use genetically engineered skin cells to inactivate cocaine and block cravings and addiction in mice.
One big challenge for gene therapies is delivering DNA or RNA safely to cells inside patients' bodies. New nanoparticles could be an improvement over the current standard – repurposed viruses.
A new research paper reports dangerous side effects in CRISPR-edited mice. Some scientists are pushing back, placing blame for the unwanted mutations on the experiment, not the technique.
Precision editing DNA allows for some amazing applications.
Ian Haydon
Researchers are starting to harness the potential of this much-hyped gene editing technique – with coming applications in medicine, biology and agriculture.
Gene therapy is growing in its capabilities, but there should be limits to its use.
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A report released by the US National Academies of Science and Medicine underscores the potential of gene editing and acknowledges the sensitivities in managing the ethical dimensions.
CF can’t currently be cured but some emerging treatments show promise.
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Cystic fibrosis (CF) affects around 3,000 people in Australia and 70,000 worldwide. It's an inherited disease caused by a mutation in a single gene called CFTR.
Honorary Senior Fellow, Department of Anatomy and Neuroscience, University of Melbourne; Associate, Centre for Health Law and Society, La Trobe University, The University of Melbourne