Gene therapy targets. Table

Antisense Oligonucleotides. Table 1 Malignant disorders as potential targets for ribozyme gene therapy... 

At the present time, almost two thirds of all gene-therapy trials target immunological and oncological diseases (Table 2). 

PCI has been shown to increase the biological activity of a large variety of macromolecules and other molecules that do not readily penetrate the plasma membrane (Table 1). PCI has also been shown to enhance the treatment effect of targeted therapeutic macromolecules. These results show that PCI can induce efficient light-directed delivery of macromolecules into the cytosol, indicating that PCI may have a variety of useful applications for site-specific drug delivery, e.g., in gene therapy, vaccination, and cancer treatment. PCI of BLM has recently been approved for a clinical Phase I trial, where several indications have been included. The first patients with head and neck cancer have recently been treated at... 

Other promoters that may be useful for the design of targeted vectors are those induced by disease-specific conditions, such as hypoxia (see Table 1). Thus, a promoter responding to hypoxia through activation by the hypoxia-inducible factor-1 (HIF-1) has been successfully used for experimental cancer gene therapy [69], Disease-specific conditions in tumor cells can also directly result from genetic alterations or from altered signaling pathways. The latter are presumably... 

Table 6.1. Some current and future targets for gene therapy...

Ever since the first clinical attempt of gene transfer more than 17 years ago, numerous monogenic (Table 7.1-2) and complex (multigenic) diseases (Table 7.1-3) were targeted with a large variety of gene therapy strategies. 

TABLE 7.1-2. Disease Targets for Gene Therapy Monogenic Diseases... 

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