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.
About 1 in 300 people in the general population carry the Tay-Sachs disease gene.
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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.
Nucleic acid vaccines use mRNA to give cells instructions on how to produce a desired protein.
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DNA and mRNA vaccines produce a different kind of immune response than traditional vaccines, allowing researchers to tackle some previously unsolvable problems in medicine.
The jab would be given twice a year.
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New payment models may mean more of the people who need these treatments can get them.
Effective delivery of PNA therapies may offer a way to treat multidrug-resistant infections and other diseases.
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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 muscula atrophy causes muscle wasting and loss of control, usually starting in infancy.
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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.
Presidential Professor and Director of Graduate Studies for biology programs; Director, Cellular & Behavioral Neurobiology Graduate Program, University of Oklahoma