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.
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Service Category |
Case |
Genetic Modification Method |
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Rare disease model |
Thrombocytosis, Temple-Baraitser Syndrome (TMBTS), Intellectual Developmental Disorder, X-Linked, Syndromic 34 (MRXS34), etc. |
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Nervous system disease model |
Autism, Paroxysmal Nonkinesigenic Dyskinesia (PNKD), Lissencephaly, etc. |
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Eye disease model |
Congenital Myopia, Wet Macular Degeneration, Familial Exudative Vitreoretinopathy (FEVR), etc. |
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Metabolic disease model |
Hypercholesterolemia, Atherosclerosis Susceptibility (ATHS), etc. |
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Gene therapy customized model |
Various gene-regulated cells, rat and mouse models |
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Type of Disease |
KO/ cKO Knockout Live Mouse |
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Amyotrophic Lateral Sclerosis (ALS) |
SOD1 |
TREM2 |
UNC13A |
CCNF |
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C9ORF72 |
OPTN |
PON2 |
ANXA11 |
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PON1 |
NEFH |
NEK1 |
SETX |
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VCP |
PPARGC1A |
GLT8D1 |
TRPM7 |
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VAPB |
EPHA4 |
GLE1 |
PARK7 |
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UBQLN2 |
CCS |
ERBB4 |
…… |
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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.
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Type |
Name |
Experimental animal |
Modeling method |
|
Mental diseases |
Depression |
Mouse |
Carotid artery ligation |
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Menopausal depression/Osteoporosis |
Mouse |
Ovariectomy (OVX) |
|
|
Cardiovascular diseases |
Aortic Sclerosis |
Mouse |
Abdominal aortic constriction |
|
Stroke |
Mouse |
（MCAO） |
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Myocardial ischemia |
Mouse |
Coronary artery ligation |
|
|
Metabolic diseases |
Liver damage |
Mouse |
Hepatic ischemia-reperfusion (HIR) |
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Renal ischemia reperfusion Injury |
Mouse |
Nephrectomy |
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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