When just one of the thousands of microRNAs in people go awry, it can cause diseases ranging from heart disease to cancer.
Genetics expert Jean Bennett explains how gene therapy is being used to treat certain forms of inherited blindness.
Chemist David Liu explains how gene editing is paving the way to treating and even curing certain genetic diseases.
At the Third International Summit on Human Genome Editing, experts gather to discuss the path forward for CRISPR and other gene-editing technologies
This technique could also be applied to other conditions, such as Parkinson’s disease.
Gene therapies and vaccines are often injected into muscle cells that are inefficient at producing desired proteins. Making them work more like liver cells could lead to better treatment outcomes.
Tay-Sachs is a rare and fatal neurodegerative disorder that most commonly affects children. Researchers have developed the first Tay-Sachs treatment to reach clinical trials.
DNA and mRNA vaccines produce a different kind of immune response than traditional vaccines, allowing researchers to tackle some previously unsolvable problems in medicine.
Gene silencing drugs target the underlying causes of a disease, rather than the symptoms it causes.
New payment models may mean more of the people who need these treatments can get them.
Antibiotic resistance is one of the biggest public health threats in the world. New research, however, may have found a way to keep up with rapidly evolving bacteria.
Spinal muscular dystrophy affects at least 1 in 10,000 people, but new drugs have given hope to those suffering from this rare disease.
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.
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.
Strategies to cure various types of blindness are looking more plausible after a series of recent breakthroughs using gene editing and gene therapy.
Genome sequencing technologies have transformed biological research in many ways, but have had a much smaller effect on the treatment of common diseases.
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.
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 for inherited retinal diseases are blossoming. Blind and partially sighted people are helping to advance the research.
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.