More than two years after its approval, only about 60 patients across the U.S., Middle East, and Europe have been treated with the gene-editing therapy. Specialists at four sickle centers told STAT ...

The efficacy of adeno-associated virus (AAV)-mediated gene therapy for the inner ear is fundamentally constrained by the natural tropism of viral ...

Scientists at the University of Virginia School of Medicine are chasing a new way to slow glioblastoma, the deadliest brain ...

A research team has successfully demonstrated the world's first gene-editing treatment for Leber's hereditary optic ...

Gene therapy holds the promise of preventing and curing disease by manipulating gene expression within a patient's cells. However, to be effective, the new gene must make it into a cell's nucleus. The ...

Gene therapy holds the promise of preventing and curing disease by manipulating gene expression within a patient's cells. However, to be effective, the new gene must make it into a cell's nucleus. The ...

Pritelivir met its primary endpoint of superior lesion healing, achieving a 62.7% lesion healing rate compared to 34.0% with standard-of-care therapies (investigator’s choice), resulting in an ...

Partner Therapeutics, Inc. (PTx), a privately held, fully integrated biotechnology company, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) ...

The new workflow was adapted from an enzymatic DNA tagging technology, DNA-TAG, previously developed in the Devaraj lab. In ...

People are suddenly very concerned about the powerhouse of the cell.

Global cholera vaccine supply has now increased to a level sufficient to allow the resumption of life-saving preventive ...