Rare diseases, defined by FDA as “a disease or condition that affects less than 200,000 people in the United States” (1), may ...

The movement or acquisition of genetic material between organisms that is not the result of vertical inheritance (reproduction) is called horizontal (or lateral) gene transfer. Horizontal gene ...

In the microscopic world of bacteria, gene transfer is a powerful mechanism that can alter cellular function, drive antibiotic resistance and even shape entire ecosystems. Now an interdisciplinary ...

The illustration shows how high trafficking of blood stem cells in the circulation early post-natally allows lentiviral-vector mediated in vivo gene transfer. “After birth, blood stem cells need to ...

This story is part of a series on the current progression in Regenerative Medicine. In 1999, I defined regenerative medicine as the collection of interventions that restore tissues and organs damaged ...

Significant unmet needs in SMA management include therapy access, fatigue, reproductive health, and emerging phenotypes in older patients. Intrathecal gene therapy offers potential for older SMA ...