Toward Tightly Tuned Gene Expression Following Lentiviral Vector Transduction

Gene Regulatable Lentiviral Vector System

Toward Personalized Gene Therapy: Characterizing the Host Genetic Control of Lentiviral-Vector-Mediated Hepatic Gene Delivery

The success of lentiviral vectors in curing fatal genetic and acquired diseases has opened a new era in human gene therapy. However, variability in the efficacy and safety of this therapeutic approach has been reported in human patients. Consequently, lentiviral-vector-based gene therapy is limited to incurable human diseases, with little understanding of the underlying causes of adverse effect...

Lentiviral vector-mediated transduction of adult neural stem/progenitor cells isolated from the temporal tissues of epileptic patients

Objective(s): Neural stem/progenitor cells (NS/PCs) hold a great potential for delivery of therapeutic agents into the injured regions of the brain. Efficient gene delivery using NS/PCs may correct a genetic defect, produce therapeutic proteins or neurotransmitters, and modulate enzyme activation. Here, we investigated the efficiency of a recombinant lentivirus vector ...

Transduction of ferret airway epithelia using a pre-treatment and lentiviral gene vector

BACKGROUND The safety and efficiency of gene therapies for cystic fibrosis (CF) need to be assessed in pre-clinical models. Using the normal ferret, this study sought to determine whether ferret airway epithelia could be transduced with a lysophosphatidylcholine (LPC) pre-treatment followed by a VSV-G pseudotyped HIV-1 based lentiviral (LV) vector, in preparation for future studies in CF ferret...

Efficient gene transduction to cultured hepatocytes by HIV-1 derived lentiviral vector.

HEPATOCYTE transplantation can be considered a viable and effective replacement therapy for enzymedeficient liver diseases as well as a supportive therapy for many forms of liver failure. However, genetic modifications of hepatocytes in an ex vivo manner may further advance the field of hepatocyte transplantation. A proofof-concept study by Grossman et al demonstrated the clinical feasibility o...