But in the 1990s the virus that causes AIDS turned out to offer a solution. Researchers discovered how to strip away HIV’s destructive genes and repurpose it as a “lentiviral vector”—a tool to deliver ...

Shares of Ultragenyx Pharmaceutical RARE were up 15.5% yesterday following the completion of the rolling submission of a ...

Scientists have developed a novel approach of genome editing to repair muscle stem (satellite) cells, offering new hope for ...

Ultragenyx (RARE) announced that it has completed the rolling submission of its biologics license application to the FDA, seeking approval for ...

Gene therapy is a promising technique for the treatment of a wide range of diseases at the genetic level, addressing the root cause of many subsequent disorders. Recent advancements have facilitated ...

Adeno-associated viruses (AAV) have emerged as pivotal delivery vehicles for gene therapy due to their overall safety (less immunogenic), efficiency (broad tropism), and long-term gene expression.

Serum CXCL6 was higher in ANCA-associated vasculitis with interstitial lung disease and independently predicted ILD ...

Several quality attributes (QAs) must be monitored to ensure an effective and safe final product when developing adeno-associated virus (AAV) vectors as drug products. Thermal stability, aggregate ...

A Navy investigation into the deadly sinking of a Marine Corps amphibious assault vehicle last year found leadership confusion, poor communications and gaps in training. But ultimately the service ...