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Different from the principles of traditional drug therapy, gene therapy regulates the expression of specific genes in the human body. Common gene therapy strategies include the introduction of normal genes (gene supplementation therapy), repair of defective genes (gene-editing therapy), and gene replacement therapy. The research of gene therapy is dependent on animal models of gene modification, surgical model, drug induction, and carrier modeling. As an international and innovative CRO platform based on research model animals, Cyagen makes the establishment of gene-edited animal models more efficient through its self-developed TurboKnockoutⓇ technology and CRISPR-Pro technology. In addition, Cyagen can provide surgical disease models and gene therapy vector model construction services.


Genetically Modified Animal Model 
Gene therapy is effective in the treatment of a variety of genetic diseases and chronic diseases. Cyagen can provide animal models for researching rare diseases, neurological diseases, ophthalmic diseases, and other customized animal models for gene therapy.


Service Category


Genetic Modification Method

Rare disease model

Thrombocytosis, Temple-Baraitser Syndrome (TMBTS), Intellectual Developmental Disorder, X-Linked, Syndromic 34

(MRXS34), etc.

Overexpression, Interference, Gene-editing

Nervous system disease


Autism, Paroxysmal Nonkinesigenic Dyskinesia (PNKD),

Lissencephaly, etc.

Eye disease model

Congenital Myopia, Wet Macular Degeneration, Familial

Exudative Vitreoretinopathy (FEVR), etc.

Metabolic disease model

Hypercholesterolemia, Atherosclerosis Susceptibility (ATHS), etc.

Gene therapy customized model

Various gene-regulated cells, rat and mouse models

Cyagen Knockout Catalog Models Case 
Cyagen Knockout Catalog Models provides over 16k research-ready gene knockout mice and conditional knockout mice. The powerful database gives you a more convenient experience. Researchers can search for their genes of interest, review the research model validation data, and submit an inquiry online. For example, there are many genes related to Amyotrophic Lateral Sclerosis (ALS), so we have developed a series of genetically modified animal models for these important disease genes for the Knockout Catalog Models. Combined with Cyagen's customized rat/mouse model development and rat/mouse breeding services, this can help you complete the task of constructing gene therapy animal models in a shorter period of time.

Type of Disease

KO/ cKO Knockout Live Mouse

Amyotrophic Lateral

Sclerosis (ALS)


























Surgical Models Case

At present, animal disease models are established through acquired modeling (such as surgical or drug induction) and then treated by AAV injection, which is used in the field of gene therapy research. Cyagen has a strong and efficient surgical disease model team, which can provide customers with a variety of surgical model services including but not limited to: mental diseases, cardiovascular diseases, and metabolic diseases. In addition, we are also actively developing new surgical disease models. We are proficient in a variety of modeling technologies, and can complete the tasks from AAV vector construction to in vivo injection according to the needs of gene therapy researchers, truly providing you with a one-stop service of high-quality experience. 




Experimental animal

Modeling method

Mental diseases



Carotid artery ligation

Menopausal depression/Osteoporosis


Ovariectomy (OVX)

Cardiovascular diseases

Aortic Sclerosis


Abdominal aortic constriction




Myocardial ischemia


Coronary artery ligation

Metabolic diseases

Liver damage


Hepatic ischemia-reperfusion (HIR)

Renal ischemia reperfusion Injury



Gene Therapy Vector Modeling:

Gene therapy vector modeling refers to local injection and systemic injection of viral vectors containing disease-causing genes to cause disease phenotypes in the whole body or part of the animal's tissues, which can be used to prepare for future treatment experiments. It is usually suitable for homozygous lethal disease models and used to model diseases in specific tissues or cells.

The hepatitis B virus (HBV) model is administered by intravenous injection, and the AAV vector is used to transport the HBV virus genome into the animal body to simulate the preparation of an animal model of HBV persistent infection. It has the advantages of high success rate, uniformity and stability, and wide application range, etc. This can greatly accelerate the development cycle of new drugs for hepatitis B treatment.

Graves' disease (GD) is an autoimmune disorder that leads to overactivity of the thyroid gland, and is the most common cause of hyperthyroidism in adults. Between 25 and 50 percent of people with Graves disease have eye abnormalities, which are known as Graves ophthalmopathy (GO). These eye problems can include swelling and inflammation, redness, dryness, puffy eyelids, and a gritty sensation like having sand or dirt in the eyes. Rarely, affected individuals have more serious eye problems, such as pain, double vision, and pinching (compression) of the optic nerve connecting the eye and the brain, which can cause vision loss. The pathogenesis is unclear. As the treatment options for GO are currently quite limited and unsatisfactory, there is an urgent need to develop new GO management strategies. Adenovirus expressing human thyroid-stimulating hormone (TSH) receptor A subunit (Ad-TSHRA) was injected into the muscle of female BALB/c mice 9 times to induce GO. Histological examination of the posterior tissue and thyroid gland at predetermined time points is performed to dynamically monitor changes, check serum autoantibodies and total thyroxine levels to assess thyroid function.

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Related Posts:

>> Viral Vector Design and Development

>> Gene Therapy Solutions

>> Effectiveness Evaluation Service

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