Gene Therapy
Motivation
Gene therapy aims to cure both inherited and acquired diseases by adding, replacing, or correcting genes through the introduction of foreign nucleic acids.In this new approach to medicine, diseases which have a genetic basis can be cured at their root, instead of just mitigating their symptoms. Obvious targets are inherited diseases, but an even more important target are diseases caused by an acquired genetic defect (e.g. cancers). In addition, many other diseases can be targeted with gene therapy, with such prominent targets as HIV/AIDS, Parkinsons and cardiovascular diseases among them.
How does it work?
For more information about Professor Safinya's research, please visit the Safinya Group Webpage.
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Selected Publications
Chan, C.-L.; Majzoub, R. N.; Shirazi, R. S.; Ewert, K. K.; Chen, Y.-J.; Liang, K. S.; Safinya, C. R.: Endosomal Escape and Transfection Efficiency of PEGylated Cationic Liposome–DNA Complexes Prepared with an Acid-Labile PEG-Lipid. Biomaterials 2012, 33, 4928-4935. DOI: 10.1016/j.biomaterials.2012.03.038.
Shirazi, R. S.; Ewert, K. K.; Leal, C.; Majzoub, R. N.; Bouxsein, N. F.; Safinya, C. R.: Synthesis and Characterization of Degradable Multivalent Cationic Lipids with Disulfide-Bond Spacers for Gene Delivery. Biochim. Biophys. Acta - Biomembranes 2011, 1808, 2156-2166. DOI: 10.1016/j.bbamem.2011.04.020.
Shirazi, R. S.; Ewert, K. K.; Leal, C.; Majzoub, R. N.; Bouxsein, N. F.; Safinya, C. R.: Synthesis and Characterization of Degradable Multivalent Cationic Lipids with Disulfide-Bond Spacers for Gene Delivery. Biochim. Biophys. Acta - Biomembranes 2011, 1808, 2156-2166. DOI: 10.1016/j.bbamem.2011.04.020.