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.
A team of Mass Eye and Ear Scientists led by Albert Edge, PhD, working with research fellow Yushi Hayashi, MD, PhD, has identified the mechanism that can lead to deafness in the rare syndrome, Norrie disease.
A team of scientists identified a means to employ gene therapy to convert glial brain cells into neurons, reinstating visual function.
When people think of DNA, they visualize a string-like double helix structure. In reality, the DNA double helix in cells is supercoiled and constrained into loops. This supercoiling and looping are known to influence every aspect of DNA activity, but how this happens has not been clear.
After several decades of pre-clinical development, cell- and gene-based therapies for Parkinson's disease (PD) are now actively being explored. In this special supplement to the Journal of Parkinson's Disease on "Repairing the Parkinsonian Brain," experts highlight some of the current strategies being pursued to restore lost function and replace what is lost in the PD brain, with special emphasis on the challenges associated with translating advanced therapeutic approaches into pioneering clinical trials.
Gliomas are the most common primary brain tumors in adults. Among them, high-grade glioblastomas (GBMs) are particularly known to be notoriously aggressive and invasive, which makes it challenging to treat them.
A compact CRISPR-Cas system, known as CasMINI, can be employed for gene-therapy and cell-engineering applications as it is easier to deliver into cells.
For the first time, Stanford researchers have miniaturized CRISPR for mammalian gene editing, which magnifies the scope of CRISPR technology.
Researchers have introduced a novel gene therapy program that could set the stage for new treatments for neurodegenerative diseases.
RNA molecules while carrying genetic instructions from DNA to the protein-making machinery of cells could help guard against non-alcoholic fatty liver.
A new study published in Nature Communications suggests that gene therapy delivered into the brain may be safe and effective in treating aromatic L-amino acid decarboxylase (AADC) deficiency.
Newly devised gene therapy can help children born with AADC, a rare genetic disorder that causes developmental and physical disabilities.
Children with a devastating genetic disorder characterized by severe motor disability and developmental delay have experienced sometimes dramatic improvements in a gene therapy trial launched at UCSF Benioff Children's Hospitals.
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.