Knockout mice have been essential for gaining proper understanding of gene function, studying human disease mechanisms, and accelerating drug development - playing an important role in biomedical advancements across a variety of research areas. With the development of gene editing technology, knockout (KO) mice become increasingly accessible for researchers worldwide. In this article, we review the basic information on knockout mice research, application examples, and the development process for custom KO mouse models.
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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. We are committed to enabling development of therapeutics for rare diseases by developing accessible animal models to study disease mechanisms, target validation, drug screening and more.
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Huntington's disease (HD) is a rare, progressive brain disorder that is inherited in an autosomal dominant manner - this disorder is caused by a defective huntingtin (HTT) protein that changes the brain, causing patients to experience problems with behavior, thinking and involuntary movements.
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Familial hypercholesterolemia (FH) is characterized by elevated levels of low-density lipoprotein cholesterol (LDL-C) and premature cardiovascular disease (CVD). FH is an autosomal dominant genetic disease (with a gene dosage effect) that is caused by mutations in genes encoding low-density lipoprotein receptor (LDLR), apolipoprotein B (APOB) or subtilisin converting enzyme 9 (PCSK9). Around 90% of FH is caused by LDLR mutations.
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Since IL17A plays an important role in infectious diseases, inflammatory, autoimmune diseases and cancer studies, IL17A has become a hot research target for many studies. In this Gene of the Week article, we have collected some insights on IL17A research progress and development trends, aiming to inspire even more scientific innovations.
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As researchers seek the most accurate animal models for in vivo experiments, the availability of gene-editing technologies for developing both mouse and rat models has created more options than ever before. Murine genomes share great similarity with the human genome, which has made them fantastic models for a wide range human disease research.
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Spinal muscular atrophy (SMA) is a genetic disease that involves the loss of motor neurons in the spinal cord, which affects the central nervous system, peripheral nervous system, and voluntary muscle movement. The primary pathogenic gene of spinal muscular atrophy is the survival of motor neuron 1 (SMN1) gene, mutations of which cause a deficiency of SMN motor neuron protein – ultimately resulting in Chromosome 5 SMA, the most common form of SMA.
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With over 300 million people worldwide living with a rare disease, Rare Disease Day aims to raise awareness of what it means to be rare. On the last day of February each year, the Rare Disease community comes together to work toward more equitable access to diagnosis, treatment, care, and social opportunity for people with a rare disease.
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The SIRT3 gene plays an important role in the pathogenesis of metabolic, cardiovascular, and neurodegenerative diseases. In this article, we review the functionality of SIRT3 and explore its role in metabolism & cardiovascular disease studies - bringing together insights to SIRT3 gene research developments to provide inspiration for your scientific innovation.
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In most experiments, scientists will try to minimize the number of independent variables so as not to confuse the results of an experiment. Due to the varying level of gene expression achieved by traditional pronuclear injection (PNI), using the offspring from a transgenic mouse created via PNI may not be the best option.
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