On August 18, 2021, the American Society of Gene and Cell Therapy (ASGCT) hosted a
virtual roundtable on adeno-associated virus (AAV) integration, featuring leading experts in …

Animal cell-based expression platforms enable the production of complex biomolecules
such as recombinant proteins and viral vectors. Although most biotherapeutics are produced …

Recent clinical trials of gene therapy have shown remarkable therapeutic benefits and an
excellent safety record. They provide evidence for the long-sought promise of gene therapy …

The growing clinical success of hematopoietic stem/progenitor cell (HSPC) gene therapy
(GT) relies on the development of viral vectors as portable" Trojan horses" for safe and …

In a range of human trials, viral vectors have emerged as safe and effective delivery vehicles
for clinical gene therapy, particularly for monogenic recessive disorders, but there has also …

Interactions between newly integrate DNA and the host genome limit the reliability and
safety of transgene integration for therapeutic cell engineering and other applications …

X-linked SCID (SCID-X1) is amenable to correction by gene therapy using conventional
gammaretroviral vectors. Here, we describe the occurrence of clonal T cell acute …

The first gene therapy clinical trials were initiated more than two decades ago. In the early
days, gene therapy shared the fate of many experimental medicine approaches and was …

Cell-based therapies are fast-growing forms of personalized medicine that make use of the
steady advances in stem cell manipulation and gene transfer technologies. In this Review, I …

γ-Retroviral vectors (γRVs), which are commonly used in gene therapy, can trigger
oncogenesis by insertional mutagenesis. Here, we have dissected the contribution of vector …