Viability of transduced motor neurons was estimated by direct counting of cells under phase contrast. A trend for reduced viability was observed with both IN− and IN+ vector types as the MOI increased ...

Cis-elements within retroviral expression constructs increase vector titer and/or gene transfer efficiency. Several approaches for expressing multiple genes in a single vector are available. The ...

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 ...