Gene therapy trials for inherited retinal diseases are blossoming. Blind and partially sighted people are helping to advance the research.
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Articles on Gene therapy
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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.
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?
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?
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”.
In a completely new approach to treating addiction, researchers use genetically engineered skin cells to inactivate cocaine and block cravings and addiction in mice.
An increasing list of rare diseases can now be treated with gene therapy. But we need to figure out a way to make them affordable.
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 successful gene therapy for the leading cause of sight loss in children is being reviewed by the FDA.
The gene therapy CTL019 induced complete remission in 90% of patients.
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.
Researchers are starting to harness the potential of this much-hyped gene editing technique – with coming applications in medicine, biology and agriculture.
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.
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.
How new therapies could prove a headache for the world of sport.
A toddler from London has had gene therapy for leukaemia and it seems to have worked. But why have breakthroughs taken so long?
Until more is understood, it’s sensible to limit experimentation that would make changes to germ line cells that would be passed on to future generations.
Gene therapy is allowing us to switch on natural beneficial mutations to counteract the effects of negative mutations in diseases such as sickle cell anaemia.
That genetic editing techniques have become as straightforward as they have poses questions for how we want them to be used.
When you buy a diet coke, or any other consumables containing the artificial sweetener aspartame, you’ll see a warning against consuming the product if you have phenylketonuria, an inherited metabolic…
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Deputy Vice-Chancellor Academic Quality and Professor of Molecular Biology, UNSW Sydney
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Researcher, L’Université d’Ottawa/University of Ottawa
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Senior Research Fellow, Melbourne Centre for the Study of Higher Education, University of Melbourne, The University of Melbourne
