Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery.
Researchers from RCSI University of Medicine and Health Sciences have developed polypeptide-based materials that serve as effective vectors in delivering gene therapies.
A preclinical study led by scientists at Sanford Burnham Prebys has established that AAV8-TNAP-D10--a gene therapy that replaces a key enzyme found in bone--may be a safe and effective single-dose treatment for hypophosphatasia (HPP).
CRISPR technology enables researchers to edit genomes by modifying DNA sequences and hence gene function.
Human genetics and genomifefcs contributed $265 billion to the U.S. economy in 2019 and has the potential to drive significant further growth given major new areas of application, according to a new report issued today by the American Society of Human Genetics (ASHG).
A gene called GAS2 plays a key role in normal hearing, and its absence causes severe hearing loss, according to a study led by researchers in the Perelman School of Medicine at the University of Pennsylvania.
Researchers from UT Southwestern used a new kind of gene therapy to successfully treat mice with Duchenne muscular dystrophy (DMD), distinctively employing CRISPR-Cas9-based tools to restore a large section of the dystrophin protein missing in several DMD patients.
In celebration of National DNA Day, AZoLifeSciences interviews renowned DNA expert Professor George Church about his life-long career in DNA research.
A gene therapy protects eye cells in mice with a rare disorder that causes vision loss, especially when used in combination with other gene therapies, shows a study published today in eLife.
A new study shows that the advantageous effects of gene therapy can be observed several decades after the body clears the transplanted blood stem cells.
The world’s first systematic study has explained how SARS-CoV-2 and other human viruses are more adapted to infect specific types of tissues..
Researchers at University of California San Diego School of Medicine have launched a first-in-human Phase I clinical trial to assess the safety and efficacy of a gene therapy to deliver a key protein into the brains of persons with Alzheimer's disease (AD) or Mild Cognitive Impairment (MCI), a condition that often precedes full-blown dementia.
A paper published today in Nature shows how chemicals in the areas surrounding tumors--known as the tumor microenvironment--subvert the immune system and enable cancer to evade attack. These findings suggest that an existing drug could boost cancer immunotherapy.
Dyno Therapeutics has demonstrated the use of AI to created an unparalleled range of adeno-associated virus capsids that can evade the immune system.
Researchers have revealed how a rare DNA change rebalanced the immune system of patients with a life-threatening genetic immunodeficiency.
For the first time, scientists have successfully used gene therapy to make mice walk again after these animals suffered a complete cross-sectional injury.
Spinal cord injury (SCI) often causes disability and seriously compromises quality of life. While decades of research have made significant progress in axonal regeneration after SCI, most of the interventions have not been translated into clinical therapies.
A new study identified an adenovirus gene therapy vector carrying a VEGF isoform. It can improve uterine blood flow in placental insufficiency, as reported in the peer-reviewed journal Human Gene Therapy.
Jose Bianco Moreira from NTNU is now convinced that gene therapy can be used to achieve the positive health benefits of physical exercise.
Researchers think they have found a fountain of youth, and it is unique to a few French Canadian families.
Researchers have created a novel gene therapy approach that shows promise for someday treating an eye disease that results in progressive vision loss.