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 ...

U.S. pharmaceutical company Eli Lilly announced on the 25th of last month (local time) interim results from Phase 1 clinical ...

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 ...

Gene editing is now reaching the mainstream, ushering in a new era of genetic manipulation. Traditionally, inserting or deleting entire genes, regulating their expression, and altering specific ...

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 ...

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 ...

There are probably around 20,000 genes in the human genome that code for protein, and cells have to transcribe DNA sequences into RNA, which is then processed before it is translated into proteins by ...