Targeted Genome Modifications Using Integrase‐deficient Lentiviral Vectors

Beta thalassemia gene therapy using lentiviral vectors

Recent years, allogeneic bone marrow transplantation (BMT) has proved to be the successful cure for patients with thalassemia major, however this is restricted due to limited matched-related donor. Its complications include chronic graft-versus-host disease in 5-8% of patients. So, a molecular approach, such as gene therapy for direct normal beta globin gene transmission, seems quite promising ...

Targeted Lentiviral Vectors: Current Applications and Future Potential

lentiviral vectors titration using real-time pcr

lentiviral vectors (lvs) are useful vehicle for genetransfer to dividing and non-dividing cells and genetic manipulations. however, the use of lentiviruses in studies requires an accurate titration technique.quantitative real-time pcr (qpcr) is a sensitive technique for the indication and quantitation of retrovirals particles. in this study, we used the qpcr for lentiviral vector titeration. th...

Lentiviral Transduction of CD34+ Cells Induces Genome-Wide Epigenetic Modifications

Epigenetic modifications may occur during in vitro manipulations of stem cells but these effects have remained unexplored in the context of cell and gene therapy protocols. In an experimental model of ex vivo gene modification for hematopoietic gene therapy, human CD34(+) cells were cultured shortly in the presence of cytokines then with a gene transfer lentiviral vector (LV) expected to transd...

Targeted transgene expression in muller glia of normal and diseased retinas using lentiviral vectors.

PURPOSE Müller glia play crucial roles in retinal homeostasis and function. Genetic modification of Müller cells by viral gene delivery would be valuable for studies of their normal physiology and roles in retinal disease states. However, stable and efficient transgene expression in Müller cells after delivery of gene transfer vectors has remained elusive. Transcriptional and transductional tar...