The adeno-associated virus (AAV)-based targeting vector has been one of the tools commonly used for genome modification in human cell lines. It allows for relatively efficient gene targeting associated with 1-4-log higher ratios of homologous-to-random integration of targeting vectors (H/R ratios) than plasmid-based targeting vectors, without actively introducing DNA double-strand breaks. In th...

A rate-determining step in gene targeting is the generation of the targeting vector. We have developed bacteriophage gene targeting vectorology, which shortens the timeline of targeting vector construction. Using retro-recombination screening, we can rapidly isolate targeting vectors from an embryonic stem cell genomic library via integrative and excisive recombination. We have demonstrated tha...

fusion of two genes at dna level produces a single protein, known as a chimeric protein. immunotoxins are chimeric proteins composed of specific cell targeting and cell killing moieties. bacterial or plant toxins are commonly used as the killing moieties of the chimeric immunotoxins. in this investigation, the catalytic domain of shiga-like toxin (a1) was fused to human granulocyte macrophage c...

Glioma, as a primary tumor of central nervous system, is the main cause of death in patients with brain cancer. Therefore, development of an efficient strategy for treatment of glioma is worthy. The aim of the current study was to develop a SRL peptide-coated dendrimer as a novel dual gene delivery system for targeting the LRP receptor, an up-regulated gene in both BBB and glioma cells. To perf...

Current treatment of solid tumors is limited by severe adverse effects, resulting in a narrow therapeutic index. Therefore, cancer gene therapy has emerged as a targeted approach that would significantly reduce undesired side effects in normal tissues. This approach requires a clear understanding of the molecular biology of both the malignant clone and the biological vectors that serve as vehic...

The technology of gene knockout is based on gene targeting, a useful technique that utilizes homologous recombination to modify the genome of a living organism primordially developed in yeast Saccharomyces cerevisiae. During this process an in vitro engineered exogenous DNA fragment is transformed into a cell in order to permanently modify targeted genomic counterpart creating a selectable and ...

The concept of orphan drugs for treatment of orphan genetic diseases is perceived enthusiastically at present, and this is leading to research investment on the part of governments, disease-specific foundations and industry. This review attempts to survey the potential to use traditional pharmaceuticals as opposed to biopharmaceuticals to treat single-gene disorders. The available strategies in...