He has pioneered the development and applications
of lentiviral vectors for gene transfer, which have become one of the most widely used tool in biomedical research and, upon recently entering clinical testing, are providing a long sought hope of cure for several currently untreatable and otherwise deadly human diseases.
The study also examines the
use of lentiviral vectors for cell delivery in treating liver diseases, a tool traditionally used in treating blood disorders.
Denning, W., Das, S., Guo, S., Xu, J., Kappes, J.C., Hel, Z. «Optimization of the transductional
efficiency of lentiviral vectors: effect of sera and polycations.»
But what's more, the transplanted cells keep producing a foreign protein slipped inside with the
help of a lentiviral vector, the kind usually used for gene therapy.
The library consists of a
pool of lentiviral vectors with roughly 73 million semi-random, 30 bp DNA barcodes that are integrated into target cells» genomes upon infection.
To sidestep the shortcomings of currently used cancer models, the Salk team harnessed the
power of lentiviral vectors to infect nondividing as well as dividing cells and ferry activated oncogenes into a small number of cells in adult, fully immunocompetent mice.
We also demonstrated that this observation was not caused incidentally by a particular vectoring system: using intraductal
injection of a lentiviral vector (Bu et al., 2009) to introduce caErbb2 to ductal epithelia, we also observed accelerated tumorigenesis in the parous group relative to the virgin group (Figure 1 — figure supplement 2).
Researchers have also developed integrase - deficient lentiviral vectors for applications that require the large genetic
capacity of a lentiviral vector but for which transient transgene expression is sufficient.
Clinical
benefits of lentiviral vectors have been observed in small trials for other diseases, including the common hematopoietic disorders sickle cell anemia and beta - thalassemia.