Although promising, gene and cell therapies can pose genotoxic risks that complicate clinical application. We describe the molecular basis of these risks, discuss tools to assess genotoxicity and ...

Adeno-associated virus (AAV) vector-mediated gene delivery was recently approved for the treatment of inherited blindness and spinal muscular atrophy, and long-term therapeutic effects have been ...

In the realm of gene therapy and genetic engineering, viral vectors have emerged as indispensable tools for delivering genetic material to cells. These modified viruses possess the ability to ...

The successes seen in a number of clinical studies on viral vector-based gene therapies (AAV, retroviral, and lentiviral vectors) are well documented, with an ever-broadening pipeline of products ...

A new variation of the CRISPR-Cas9 gene editing system makes it easier to re-engineer massive quantities of cells for therapeutic applications. The approach, developed at Gladstone Institutes and UC ...