Preclinical studies show that gene therapy can improve muscle strength in small- and large-animal models of a fatal congenital childhood disease know as X-linked myotubular myopathy.
The findings, appearing as the cover story in the January 22, 2014 issue of Science Translational Medicine, also demonstrate the feasibility of future clinical trials of gene therapy for this devastating disease. (more…)
The potential of gene therapy to boost heart muscle function was explored in a recent University of Washington animal study. The findings suggest that it might be possible to use this approach to treat patients whose hearts have been weakened by heart attacks and other heart conditions.
Michael Regnier, UW professor and vice chair of bioengineering, Charles Murry, director of the Center for Cardiovascular Biology and co-director of the Institute for Stem Cell and Regenerative Medicine, and Sarah Nowakowski, a UW graduate student in bioengineering, led the study. The findings appeared online March 25 in the Proceedings of the National Academy of Sciences. (more…)
AUSTIN, Texas — Researchers at The University of Texas at Austin and the Stanford University School of Medicine have found a novel way to engineer key cells of the immune system so they remain resistant to infection from HIV, the virus that causes AIDS. (more…)
UCLA stem cell researchers have found that a gene therapy regimen can safely restore immune systems to children with so-called “bubble boy” disease, a life-threatening condition that if left untreated can be fatal within one to two years.
In the 11-year study, researchers were able to test two therapy regimens for 10 children with ADA-deficient severe combined immunodeficiency (SCID), which has come to be known as “bubble boy” disease because some of its victims have been forced to live in sterile environments. (more…)
New research from North Carolina State University finds that gold nanoparticles with a slight positive charge work collectively to unravel DNA’s double helix. This finding has ramifications for gene therapy research and the emerging field of DNA-based electronics.
“We began this work with the goal of improving methods of packaging genetic material for use in gene therapy,” says Dr. Anatoli Melechko, an associate professor of materials science and engineering at NC State and co-author of a paper describing the research. Gene therapy is an approach for addressing certain medical conditions by modifying the DNA in relevant cells. (more…)
Researchers at Yale University have developed a novel nanoparticle with promising applications in gene therapy, a type of medical treatment that addresses the root causes of diseases now typically treated for symptoms.
The advance could lead to new therapies for many forms of cancer, including brain tumors, as well as for cystic fibrosis and Huntington’s Disease. (more…)
COLUMBUS, Ohio – For the first time, researchers have found a way to inject a precise dose of a gene therapy agent directly into a single living cell without a needle.
The technique uses electricity to “shoot” bits of therapeutic biomolecules through a tiny channel and into a cell in a fraction of a second.
L. James Lee and his colleagues at Ohio State University describe the technique in the online edition of the journal Nature Nanotechnology, where they report successfully inserting specific doses of an anti-cancer gene into individual leukemia cells to kill them. (more…)
Researchers from North Carolina State University, the Oak Ridge National Laboratory and CFD Research Corporation have found a new way to develop straight carbon nanofibers on a transparent substrate. Growing such nanofiber coatings is important for use in novel biomedical research tools, solar cells, water repellent coatings and others. The technique utilizes a charged chromium grid, and relies on ions to ensure the nanofibers are straight, rather than curling – which limits their utility.
“This is the first time, that I know of, where someone has been able to grow straight carbon nanofibers on a clear substrate,” says Dr. Anatoli Melechko, an associate professor of materials science and engineering at NC State and co-author of a paper describing the research. “Such nanofibers can be used as gene-delivery tools. And a transparent substrate allows researchers to see how the nanofibers interact with cells, and to manipulate this interaction.” (more…)
