Gene-infused nanoparticles used for combating disease work better when they include plant-based relatives of cholesterol, new research shows. That’s because the shape and structure of the phytosterols help the genes get where they need to be inside cells. The findings by Oregon State University researchers, published today in Nature Communications, are important because many illnesses that
If approved, 1st Gene Therapy in U.S. for the Treatment of Any Type of Hemophilia BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application (BLA) to the FDA for its investigational AAV5 gene therapy, valoctocogene roxaparvovec, for adults with hemophilia
Neel Anand, MD, serves as professor of orthopedic surgery and director of spine trauma at the Cedars-Sinai Spine Center in Los Angeles. Dr. Anand has dedicated his practice to the advancement of minimally invasive spine surgery and served as principal investigator in several FDA clinical trials. Here, Dr. Anand discusses the hype around stem cells and
Industry leaders, patient advocates, researchers unite to maximize the incredible potential of transformative gene therapies The Institute for Gene Therapies (IGT) launched today with a focus on advocating for a modernize the U.S. regulatory and reimbursement framework so that gene therapies can deliver their significant potential to patients. IGT will educate stakeholders across the healthcare
By carefully harvesting tiny samples of olfactory cleft mucosal tissue and conducting single-cell analysis, a collaborative team led by University of Miami Miller School of Medicine researchers has found compelling evidence to support olfactory neurogenesis, the ability to regrow olfactory neurons to the brain. “For decades, medical researchers have wondered whether or not neurogenesis was
Within the framework of a European funded H2020 project, VANGUARD aims to develop a cure for type 1 diabetes available to a larger group of patients. The VANGUARD project will use advanced tissue engineering techniques to generate a bioartificial pancreas, with the aim to propose a novel cell-based treatment to type 1 diabetes that overcomes
Korean researchers have successfully demonstrated knee cartilage regeneration by using stem cells delivered by a magnet-controlled microrobot, raising hope to broaden a non-invasive procedure for patients who need invasive knee replacement surgery. The new method can also raise stem cell delivery accuracy efficiency by up to 95 percent compared to less than 20 percent in
Bill Gates calls for reducing health care inequities in a AAAS Annual Meeting plenary address. The number of child deaths around the world has declined by half in the past two decades, but that number could be cut even further with the help of artificial intelligence and gene editing tools, said Bill Gates at the
A team of scientists from Purdue University and institutions around the world have come together to better understand the growing number of worldwide patented innovations available for gene therapy treatment. The global gene therapy market is expected to reach $13 billion by 2024 as new treatment options target cancers and other diseases. Now, a team of scientists
In the first clinical study of its kind in the US, researchers used CRISPR to modify CAR T cells to make them more potent against cancer, but the clinical benefits are unknown. mmune cells altered by CRISPR are safe to use in cancer patients, according to results of a clinical trial reported February 6 in Science.
