In seven patients with Danon disease, a single infusion of adeno-associated virus serotype 9 carrying transgene LAMP2B ...

Ten years ago, nobody knew that Asgard archaea even existed. In 2015, however, researchers examining deep-sea sediments ...

Notably, no participant developed anti-drug antibodies (ADA), a common challenge observed in other gene-based delivery platforms, such as adeno-associated virus (AAV) mediated antibody expression ...

A gene therapy to correct a mutation that causes maple syrup urine disease prevented newborn death and normalized growth in a calf as well as in mice.

ChromaTan Inc. and Landmark Bio PBLLC have been awarded a grant from the National Institute for Innovation in Manufacturing ...

Liver injury is a known complication risk of Elevidys and other gene therapies that use an adeno-associated (AAV) virus to deliver the one-time treatment to cells. That risk is already flagged on ...

The researchers zeroed in on mAb 3A6 because it appears to target a structure on the Ebola virus called the "stalk." The stalk is an important part of the Ebola virus structure because it anchors ...

A newly discovered bat coronavirus, HKU5-CoV-2, similar to the virus that caused the COVID-19 pandemic, has been identified in China. The discovery was made by a team of virologists led by Shi ...

Rocket's first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition. Fintel is one of the most ...