In a major advancement in the field of gene therapy for rare and devastating diseases, researchers at Children’s Hospital of Philadelphia (CHOP) have developed a “dimmer switch” system that can control levels of proteins expressed from gene therapy vectors. The system is based on alternative RNA splicing using an orally available small molecule and works effectively in tissues throughout the body, including the brain. The first research regarding this innovation was published today in the journal Nature.
Tag: Pharmaceutical Biotechnology
Scientists Recruit New Atomic Heavyweights in Targeted Fight Against Cancer
Researchers from Berkeley Lab and Los Alamos National Laboratory have developed new methods for the large-scale production, purification, and use of the radioisotope cerium-134, which could serve as a PET imaging radiotracer for a highly targeted cancer treatment known as alpha-particle therapy.
Biden focuses on health care, but pharma firms have upper hand
President-elect Joe Biden began transition planning with the announcement of a COVID-19 task force, continuing a focus on health care that was a hallmark of the Obama-Biden era, culminating in the passing of the Affordable Care Act. Colleen Carey,…