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.
Delivering genetic material is a key challenge in gene therapy.
Invitation image created by Kstudio
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.
DNA: the new battleground.
The gene therapy CTL019 induced complete remission in 90% of patients.
Laboratory mice are among the first animals to have their diseases treated by CRISPR.
tiburi via Pixabay.com
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.
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.
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.
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.
And a little more of that one…
How new therapies could prove a headache for the world of sport.
The accuracy of gene editing will be the deciding factor in the success of current technology.
Genes by Shutterstock
A toddler from London has had gene therapy for leukaemia and it seems to have worked. But why have breakthroughs taken so long?
The red Cas9 nuclease protein uses a blue guide RNA sequence to cut yellow DNA at a complementary site.
CRISPR-Cas9 via www.shutterstock.com
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.
Genetic therapy might be able to reverse the harmful effects of sickle cell anaemia.
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.
In the future, our DNA could be different by design.
DNA by Seamartini Graphics/www.shutterstock.com
That genetic editing techniques have become as straightforward as they have poses questions for how we want them to be used.
An excess of the one of the two amino acids in the artificial sweetener aspartame is a significant concern for people with phenylketonuria.
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…
Viruses like this one may yet become a benevolent force in modern medicine.
Viruses have traditionally been mankind’s enemies, causing disease and often mutating out of the reach of our medicines. But now a new technology is conscripting them into doing good. Viral vectors show…
Increasing the activity of enzyme NEU1 can stop or reverse the build-up of plaque in the brains of mice with Alzheimer’s…
It’s hard to replace plain flour with self-raising flour in a cake, but luckily, our chromosomes are more like cars.
Every now and again you might read about gene therapy and efforts to correct serious genetic diseases. But I’m betting that very few readers have had gene therapy, nor have they ever met anyone who has…
Helpful viruses get protected by decoy viruses in the bloodstream.
The presence of foreign objects, like viruses, in our bloodstream is usually a bad thing. Evolution has created some extremely efficient immune cells that patrol the blood, seeking out material that should…
Gene therapy: not just for mice.
Genetic traits like a bulbous nose or balding give some people reasons to moan about what they inherited from their parents. But more serious genetic flaws can cause debilitating disease. Now, Italian…
The new treatment of inherited blindness in dogs might help restore vision to humans too. A combined study lead by Michigan…