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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The FGF21 gene plays a significant role in the pathogenesis of metabolic diseases such as non-alcoholic fatty liver disease (NAFLD), obesity, and insulin resistance. In this article, we review the FGF21 gene, a pathogenic gene of metabolic diseases, hoping to give you insights on FGF21 and its role in metabolic disorders.
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Duchenne muscular dystrophy (DMD) is a genetic disorder characterized by progressive muscle degeneration. It usually starts in early childhood. In this article we review the background information and research insights of the dystrophin (DMD) protein-coding gene, a pathogenic gene of Duchenne Muscular Dystrophy.
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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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Cell line-derived tumor xenograft (CDX) models play an important role in basic research of tumorigenesis and metastasis, as well as drug development and treatment. Therefore, establishing a precise animal model is vital for accurate in vivo tumor studies and in advancing oncology research.
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Alzheimer’s disease (AD) maintains the highest prevalence and morbidity among neurodegenerative diseases. As the FDA approved the first-of-its-kind treatment for Alzheimer’s disease in June 2021, Alzheimer’s drug development has come into the spotlight.
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