Positive clinical trial results on gene transfer to treat macular degeneration
The long-term safety of gene transfer to treat neovascular age-related macular degeneration (NVAMD), and the production of two therapeutic proteins encoded by those genes for at least 2.5 years in the...
View ArticleGene therapy shows promise for treating Niemann-Pick disease type C1
For the first time, National Institutes of Health (NIH) researchers have demonstrated in mice that gene therapy may be the best method for correcting the single faulty gene that causes Niemann-Pick...
View ArticleA common heart problem caused by cancer therapy avoided blood vessel treatment
Researchers at the Wihuri Research Institute and the University of Helsinki, Finland, have found that some of the harmful effects of a commonly used cancer drug can be alleviated by using gene therapy...
View ArticleBiologists identify the switch for Neuroglobin gene—a new target for...
A study led by Dr K.C. Tan-Un and her team at the School of Biological Sciences, the University of Hong Kong (HKU), in collaboration with Professor Richard Festenstein at the Clinical Sciences Centre,...
View ArticleResearcher develops safer gene therapy
A Washington State University researcher has developed a way to reduce the development of cancer cells that are an infrequent but dangerous byproduct of gene therapy.
View ArticleResearchers take step toward gene therapy for sickle cell disease
A team of researchers at the Stanford University School of Medicine has used a gene-editing tool known as CRISPR to repair the gene that causes sickle cell disease in human stem cells, which they say...
View ArticleGenetically engineering disease-fighting cells: New technique improves the...
The human body produces T cells to recognize and fight disease. Each T cell has a unique T cell receptor (or TCR) on its surface that surveils small fragments of proteins presented by other cells. Upon...
View ArticleMonocyte gene expression signatures predict how RA patients respond to...
Distinct gene expression signatures in rheumatoid arthritis patients could help rheumatologists predict how these individuals will respond to tumor necrosis factor inhibitors, and may one day enable a...
View ArticleNovel mapping technique targets gene therapy to hibernating heart muscle
Gene therapy to repair damaged heart muscle is most likely to succeed if it can be injected at the site of ischemia where there is viable myocardium with reduced contractile ability, and a new...
View ArticleUK experts give green light to 'three-parent babies'
British scientists on Wednesday approved the use of so-called "three-parent baby" fertility treatments, paving the way for the country to become the first in the world to officially introduce the...
View ArticleAfter gene therapy, hemophilia B patients maintain near-normal levels of...
Researchers are reporting the highest and most sustained levels to date of the essential blood-clotting factor IX in patients with the inherited bleeding disorder hemophilia B. After receiving a single...
View ArticleRepurposed drugs may offer improved treatments for fatal genetic disorders
University of Rochester Medical Center researchers believe they have identified a potential new means of treating some of the most severe genetic diseases of childhood, according to a study in PLOS...
View ArticleCRISPR screening identifies potential HIV treatment targets
Investigators from Whitehead Institute, the Ragon Institute of MGH, MIT and Harvard and the Broad Institute of MIT and Harvard have used CRISPR-Cas9 gene-editing technology to identify three promising...
View ArticleGene therapy to prevent vision loss: Studies in primates needed to optimize...
Many gene therapy-based approaches are in development to combat genetic and other causes of blindness and vision loss, and much can be learned about the safety and effectiveness of these promising new...
View ArticleGene therapy for liver disease advancing with the help of adeno-associated...
Liver-directed gene therapy delivered using adeno-associated viral (AAV) vectors to treat diseases such as hemophilia have advanced into human testing. The potential for continued technological...
View ArticleImmunotherapy, gene therapy combination shows promise against glioblastoma
"Devastating" and "dismal." That's how leading researchers describe the present outlook for malignant brain tumors. The median survival rate for patients with glioblastoma multiforme, or GBM, is a mere...
View ArticleThe science of replacing mitochondrial DNA and what remains unknown
Mitochondrial replacement therapy (MRT) has now been used in humans to conceive a "three-parent baby" to prevent inherited mitochondrial disorders, but there remain questions about the effectiveness of...
View ArticleGene delivery to the lung can treat broad range of diseases within and beyond...
Targeting therapeutic genes to the lungs offers the potential to manage serious lung diseases that do not respond to other forms of treatment and to use the lungs as metabolic factories to produce...
View ArticleGene therapy for pompe disease effective in mice, poised for human trials
After decades investigating a rare, life-threatening condition that cripples the muscles, Duke Health researchers have developed a gene therapy they hope could enhance or even replace the only...
View ArticleNew gene-delivery therapy restores partial hearing, balance in deaf mice
Using a novel form of gene therapy, scientists from Harvard Medical School and the Massachusetts General Hospital have managed to restore partial hearing and balance in mice born with a genetic...
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