The availability of gene-editing technologies for developing rodent models has created more options for researchers than ever before. Murine genomes share significant similarities with the human genome, which has made them fantastic models for a wide range of human disease research. Choosing suitable animal models is essential for researchers to study the mechanisms of human disease and evaluate drug efficacy.
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To understand disease mechanisms and explore effective therapeutic targets, the selection of experimental models is essential. In this on-demand Webinar, Dr. Marvin Ouyang, Executive Vice President and Chief Scientific Officer of Cyagen, explains the current research progress on rare diseases, introduces successful cases of using animal models in rare disease therapeutic research and effective gene-editing strategies that can accelerate your research progress!
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The World Health Organization (WHO) defines rare diseases as diseases with a total population of 0.65-1 %. Due to a low incidence rate, rare diseases are also known as "orphan diseases", and the corresponding drugs are called orphan drugs. Although the number of patients with each disease is small, the total number of rare disease patients is very large due to the wide variety of diseases. There are more than 300 million patients living with rare diseases in the world. Thus, it is of great significance to develop treatments for rare diseases.
Since about 80% of the rare diseases are caused by gene mutation, they are ideal targets for gene therapy treatment. This article will introduce the current research progress and case analysis of gene therapies for rare diseases.
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Gene therapy refers to the introduction of exogenous (normal) genes into target cells to correct or compensate for the diseases caused by defects and abnormal genes, to achieve the purpose of disease treatment. At present, it has shown great potential in the treatment of cancer, genetic diseases such as thalassemia, sickle anemia, hemophilia, and congenital amaurosis.
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Apoe knockout mice are widely used to study the function of APOE in atherosclerosis, lipid metabolism, and nerve damage. These mice also help to study interventional therapies that can change the atherosclerotic process. In this article, we review the phenotype of APOE knockout (KO) mice and explore its applications in cardiovascular and respiratory disease research. Read on for a comprehensive introduction of how APOE knockout mice are being used as genetically engineered mouse models of disease.
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Gene therapy is designed to treat diseases by introducing exogenous DNA or RNA into human target cells, which may be achieved by a few different methods. It is used to treat inherited genetic disorders: to correct or compensate for the patient’s abnormal gene, or to silence or inhibit the abnormal expression of genes. In addition to the treatment of genetic diseases, gene therapy is also widely used in the treatment of acquired diseases, such as tumors, autoimmune diseases, organ transplantation, and more.
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With the rapid development of human antibody production, using gene editing technology to construct humanized mice with human antibodies has become an important subject in antibody drug discovery. However, due to the large genomic region of human antibody Ig genes, it makes the construction of humanized mice that express the human antibody tremendously challenging. Accordingly, it remains difficult to achieve large-fragment human antibody gene insert in animal models, especially for gene that is greater than 1 Mb.
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The continuous development and improvement of gene editing technology has driven the successful establishment of such a platform for mouse models expressing human antibody genes. This has not only led to revolutionary innovations in the research and development of therapeutic antibody drugs, but has also served to promote their wide clinical applications.
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To reduce the immunogenicity of mouse antibodies, chimeric antibody and human antibody strategies have been developed and quickly become important technologies in the antibody drug research. The establishment of phage display technology allows researchers to successfully screen and obtain the first fully human antibody with high affinity.
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The therapeutic antibody has become a leading treatment option for cancers and other related diseases. In the past 25 years, antibody therapy has become an important treatment method for various diseases, such as cancer. Notably, from 2018 to 2019, there were about 18 new therapeutic antibody drugs approved for clinical use.
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