These findings are the first to confirm the applicability of LPC pretreatment in the production of extensive lentiviral gene transfer in mouse lung airways. However, improved methodologies to increase transduction efficiency are required for adult sheep lung. The results suggest that continued in vivo development of LPC-enhanced lentiviral gene transfer is needed in the lungs of large animals to establish effective lentiviral gene transfer techniques suited to the treatment of airway disease.

Methylmalonic aciduria is a rare disorder of organic acid metabolism with limited therapeutic options, resulting in high morbidity and mortality. Positive results from combined liver/kidney transplantation suggest, however, that metabolic sink therapy may be efficacious. Gene therapy offers a more accessible approach for the treatment of methylmalonic aciduria than organ transplantation. Accordingly, we have evaluated a lentiviral vector-mediated gene transfer approach in an in vivo mouse model of methylmalonic aciduria. A mouse model of methylmalonic aciduria (Mut -/