Authors explore complexities in scaling gene-therapy production with special attention is to monitoring and enhancing ...

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 ...

The great hope for gene therapy is that one day it will be out of the clinic and into mainstream therapeutics. Bearing enormous potential to treat or cure disease, such technology can replace a ...

A 3D illustration of a transfer RNA molecule hovering over a messenger RNA. Transfer RNA acts as an adaptor to convert messenger RNA codons into amino acids during protein synthesis. Credit: ...

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 ...

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 ...

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 ...

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 ...

As we continue our series exploring genetic medicine, both in this story and in Destiny's Child No Longer: Rewriting Genetic Fate, gene therapy stands at a crossroads. On June 16, Sarepta Therapeutics ...