Engineering retroviruses into vectors of therapeutic value has been a highly pursued effort. To date, the promise of retroviral-mediated gene delivery to treat or even cure genetic diseases has been ...
Recombinant adeno-associated virus (AAV) vectors are predominantly nonintegrating, but rare genomic integration events have been associated with oncogenesis in neonatal murine models. Here we report a ...
The location of integration of a gene therapy has been crucial for the safety and efficacy of the treatment to cure infants with X-linked severe combined immunodeficiency. Identified integrome ...