Nanoparticle-directed tissue-specific delivery system for genes and drugs.
Extract: Gene therapy is recognized as a promising approach for the treatment of serious diseases including monogenic diseases, infectious diseases, and cancer. Conventional vectors used for delivering therapeutic genes are virus-based vectors, such as the adenovirus, retrovirus, lentivirus, and adeno-associated virus. Since these viruses can infect a wide range of tissues, it is essential to be able to deliver these viruses specifically to the target tissues in vivo. If these viruses are administrated intravenously, the genes will be introduced into unexpected tissues, such as those around the injected place, causing side effects. These situations also make the repetitive administration of genes by conventional virus vectors nearly impossible. Thus, the in vivo targeting of therapeutic genes is now considered as an important issue for the next generation of gene therapy. In addition, conventional virus vectors possess an upper limitation on the size of the therapeutic gene and sometimes introduce parts of the viral genome into the patient's chromosome. Moreover, the production of a large amount of virus vectors is dangerous for manufacturers, because of its high and nonspecific infectivity. This will significantly increase the production cost of viral vectors.
Kuroda
Shun-Ichi
S
Department of Structural Molecular Biology, Institute of Scientific and Industrial Research, Osaka University, 8-1 Mihogaoka, Ibaraki, Osaka 567-0047, Japan.
Yamada
Tadanori
T
Tanizawa
Katsuyuki
K