RNA interference using small interfering RNAs (siRNAs) has become a mainstay of functional gene characterization and has ...

Two poster presentations at ACNP highlight new research on the fundamental biology of molecules targeting synaptic function in CNS disease Insights advance translational model supporting Gate’s lead ...

Drugs that are taken orally need to pass through the lining of the gastrointestinal (GI) to be taken up. Transporter proteins found on cells that line the GI tract help with this process, but for many ...

Researchers identified a retinal disease to evaluate the success of gene and cell replacement therapy. In the retinas of human eyes, the cones are photoreceptor cells responsible for color vision, ...

Researchers at the Nora Eccles Harrison Cardiovascular Research and Training Institute, University of Utah, and the University of Utah School of Medicine, have demonstrated that a gene therapy can ...

Michael Gotthardt at the Max Delbrück Center and collaborators are developing a drug to treat a common type of heart failure characterized by impaired cardiac filling. In “Cardiovascular Research,” ...