Luigi Naldini

In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector.

A retroviral vector system based on the human immunodeficiency virus (HIV) was developed that, in contrast to a murine leukemia virus-based counterpart, transduced heterologous sequences into HeLa cells and rat fibroblasts blocked in the cell cycle, as well as into human primary macrophages. Additionally, the HIV vector could mediate stable in vivo gene transfer into terminally differentiated neurons. The ability of HIV-based viral vectors to deliver genes in vivo into nondividing cells could increase the applicability of retroviral vectors in human gene therapy.

Lentiviral Vectors for Gene Delivery in the Nervous System

Gene therapy is an attractive approach for the treatment of a number of inherited and acquired disorders of the nervous system. For instance, genetic manipulations could be used to provide locally the neurotransmitters that are defective in conditions such as Parkinson’s disease, or to produce healing levels of growth factors to protect neurons against the irreversible consequences of traumatic or degenerative lesions.