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

Gene transfer agents (GTAs) are unique, phage‐like particles produced by various Alphaproteobacteria that mediate the horizontal transfer of genetic material. Unlike conventional bacteriophages, GTAs ...

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 ...

Entrepreneurs say it’s time to safety-test designer baby technology. A West Coast biotech entrepreneur says he’s secured $30 million to form a public-benefit company to study how to safely create ...

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 ...

A new gene-editing technique enables the correction of multiple genetic mutations simultaneously, transforming the prospects for millions living with complex inherited diseases, such as cystic ...