Gene targeting in mice is a powerful tool for identifying the in vivo functions of proteins and for producing new animal models of human diseases. In the conventional approach, the gene encoding a protein of interest is disrupted by homologous recombination in embryonic stem (ES) cells. Targeted ES cells are injected into blastocysts to produce chimeric mice, which are then bred to produce knoc...

1. Abstract 2. Introduction 3. Hepsin cDNA and gene structures 3.1. Cloning of hepsin cDNA 3.2. Hepsin gene structure 3.3. The promoter sequence of the mouse hepsin gene. 4. Hepsin protein 4.1. The domain structure of hepsin 4.2. Biochemical properties of hepsin 4.2.1. Molecular weight 4.2.2. Membrane topology 4.2.3. Substrate specificity 4.2.4. Effect of inhibitors 5. Functions of hepsin 5.1. ...

Precise modification by gene targeting (GT) provides an important tool for studies of gene function in vivo. Although routine with many organisms, only isolated examples of GT events have been reported for flowering plants. These were at low frequencies precluding reliable estimation of targeting efficiency and evaluation of GT mechanisms. Here we present an unambiguous and straightforward syst...

The development of designed site-specific endonucleases boosted the establishment of gene targeting (GT) techniques in a row of different species. However, the methods described in plants require a highly efficient transformation and regeneration procedure and, therefore, can be applied to very few species. Here, we describe a highly efficient GT system that is suitable for all transformable pl...

The ability to specifically modify DNA sequences in the genome of mice through gene targeting has made the mouse a powerful and indispensable experimental tool for advancing biological and medical knowledge. Such genetic modifications can include for example: (i) the deletion of a gene of interest or insertion of mutations in order to determine the function of specific genes; and (ii) genetic m...

1. Abstract 2. Introduction 3. Biochemistry 3.1. Structure of the FX Protein 3.2. Structure of the FX Gene 3.3. Activation of FX 3.4. Activities of FX 3.4.1. Hemostasis 3.4.2. Non-hemostatic Functions 4. Factor X-Deficient Mice 4.1. Construction of the FX Gene Deletion 4.2. Viability of FX-Deficient Neonates 4.3. Viability and Survival of FX-Deficient Embryos 5. Perspectives 6. Acknowledgement ...

the use of animal models in modeling of human genetic disease has many advantages. in some cases, however, this method may not be applicable due to some limitations, such as differences in tissue composition, anatomy and physiology of humans and animals. isogenic human disease models are a population of cells that are selected or engineered to model a specific genetic disease, in vitro. they ar...

Gene targeting in most of human somatic cell lines has been labor-intensive because of low homologous recombination efficiency. The development of an experimental system that permits a facile evaluation of gene targeting efficiency in human somatic cell lines is the first step towards the improvement of this technology and its application to a broad range of cell lines. In this study, we utiliz...

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...

We evaluate the effect of target site transcription on gene targeting in cultured human fibrosarcoma cells. A number of cell lines that harbored a plasmid recombination substrate within their chromosomal DNA were created. Gene targeting frequency was then measured at these different loci in the presence and absence of an agent that stimulated target site transcription. We observed that gene tar...