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Entering this century, soon after the completion of the human genome project, the mouse genome was also completed. NIH embarked on the next endeavor to coordinate the knockout of every mouse gene and then study their phenotype systematically. These efforts have contributed enormously to the understanding of gene functions as well as genetic causes of human diseases. The first two decades of this century have seen tremendous progress in understanding human diseases through manipulation of the mouse genome, including knockout, conditional knockout, transgenic, knockin, point mutation, and different levels of humanization. The advent of CRISPR technology has also greatly facilitated the genetic engineering of the mouse genome. Now we can link a multitude of genetic mutations on thousands of individual genes to thousands of monogenic human diseases, a majority of which are so-called rare diseases. In this white paper, we review the gene therapy studies and humanized mouse models of genetic diseases.
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Cyagen One-Stop Gene Therapy Model SolutionsCyagen provides comprehensive model generation services for all your research needs. Contact us to perform your entire project - from initial strategy design and experimental model construction, all the way through supporting services – we deliver research-ready models and professional services for guaranteed results. Cyagen One-stop Solution for Rare Disease Drug DevelopmentMore than 80% of rare diseases are caused by specific gene mutations. Generally speaking, there are hundreds of gene mutations associated with each disease. Developing the desired animal research model via gene-editing technology requires extensive identification and phenotype analysis efforts. However, rare diseases historically have less than a 15% probability of successfully recapitulating the human phenotype in mouse models. This has posed a huge challenge for rare disease drug research & development worldwide. As a leading provider of custom mouse and rat models, Cyagen aims to support the advancement of rare disease and related gene therapy research with our expertise. Cyagen is committed to enabling development of therapeutics for rare diseases by developing precise animal models to study disease mechanisms, target validation, drug screening and more. Based on Cyagen’s innovative technology, our team can rapidly construct various types of genetically modified mouse models including gene knockout, conditional knockout, humanization, point mutation, and more. We can create any custom mouse or rat model with guaranteed genotype validation along with phenotype analysis and other downstream services to establish an accurate rare disease model. In addition, we have established a drug screening animal platform for cancer, immunology, endocrinology, cardiovascular, neurology, and infectious diseases. This platform provides high-quality and stable animal models services to support drug efficacy evaluations for CRO institutions and drug R&D institutions to accelerate the progress of drug development. In addition to the construction of mouse models, we also have rich expertise in rat model construction. So far, we have delivered over 78,400 murine models to researchers worldwide and received over 4,750 peer-reviewed citations for our products and services. Cyagen prides itself on its premium customer service: including price-matching, client access to complimentary technical consultations, full confidentiality, and a 100% money-back service guarantee. Cyagen can provide accurate animal models and comprehensive supporting services for your research. Contact us for a complimentary consultation and quote for your research model project.
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