Genetically Engineering Model Development Services
In the field of biomedical research, the development of accurate and reliable animal models is crucial for understanding the mechanisms underlying rare diseases and developing effective therapeutic strategies. Genetically Engineering Model (GEMs) development has emerged as a powerful tool in this endeavor. At our company, we are at the forefront of GEM development, providing cutting-edge services to support rare disease research and drug discovery.
Introduction to Genetically Engineering Models (GEMs)
Genetically engineering models (GEMs) refer to animal models that have been genetically modified to simulate human diseases. These models are created by introducing specific genetic alterations into the animal's genome, such as gene mutations, deletions, or insertions. The most frequently used species for such modifications are mice and rats. However, advancements in transgenic technology have enabled the creation of genetically engineered zebrafish, pigs, and even non-human primates over the past decade.
Fig.1 Schematic representation of mouse models in coloretal cancer. (Kucherlapati, M. H., 2023)GEMs for Rare Diseases
GEMs provide researchers with a controlled and reproducible system to study the pathophysiology and therapeutic interventions for various rare diseases. By using GEMs that accurately represent the disease condition, scientists can assess the effectiveness of novel drug candidates, optimize dosage regimens, and predict potential adverse effects. The table below lists the applications of genetic engineering models in corresponding rare diseases.
Table 1 Genetically engineering model (GEMs) for rare diseases. (Phan, H.T.L., Kim, K., et al., 2023)
| Gene Editing Tool | Target Gene | Human Disease Models | Mutation Type | Manipulation Methods |
|---|---|---|---|---|
| CRISPR/Cas9 | Fah | Tyrosinaemia | Knockout | Microinjection |
| CRISPR/Cas9 | FVII | Hemophilia | Knockout | Microinjection |
| CRISPR/Cas9 | Notch3 | Lateral meningocele syndrome | Knockout | Microinjection |
| CRISPR/Cas9 | Fah and Hpd | Tyrosinaemia type I | Knockout | Hydrodynamic injection |
| TALEN | Fus | Amyotrophic lateral sclerosis | Knockin | Microinjection |
| CRISPR/Cas9 | BRIL | Osteogenesis imperfecta (OI) type V | Knockin | Microinjection |
| CRISPR/Cas9 | HTT | Huntington's disease (HD) | Knockin | Microinjection |
| TALEN | HBG | β-thalassemia and sickle cell disease | Humanized | Transplantation/Engraftment |
| CRISPR/Cas9 | DMD | DMD | Humanized | Microinjection |
Our Services
Genetically Engineering Model (GEMs) development has revolutionized rare disease research, providing scientists with powerful tools to study disease mechanisms, identify therapeutic targets, and evaluate rare disease therapies. Our Company is committed to supporting rare disease research through our expertise in GEM development, providing customized models, phenotypic characterization, drug safety evaluation, and pharmacokinetics services.
- Animal Species for Model Development
-Mouse
-Rat
-Rabbit-Pig
-Dog-Monkey
-Cat-Zebrafish
-Others - Methods of GEMs Development
- Types of GEMs Development
- Circulatory System Disease Development Service
- Digestive System Disease Development Service
- Nervous System Disease Development Service
- Respiratory System Disease Development Service
- Urinary System Disease Development Service
- Endocrine System and Metabolic Disease Development Service
- Immune System Disease Development Service
- Motor System Disease Development Service
- Integumentary System Development Service
- Ophthalmological Disease Development Service
- Infectious Disease Development Service
- Rare Cancer Development Service
Why Choose Us?
At our company, we are proud to have a team of exceptional scientists who possess extensive expertise and training in genetically engineering model (GEMs) development for rare disease research. Our company is committed to delivering high-quality results and providing effective communication and problem-solving solutions to meet the evolving requirements of your rare disease research projects. If you are interested in our services, please feel free to contact us for more details and quotation information of related services.
References
- Kucherlapati, M. H., "Mouse models in colon cancer, inferences, and implications." Iscience (2023).
- Phan, H.T.L., Kim, K., et al., Progress in and Prospects of Genome Editing Tools for Human Disease Model Development and Therapeutic Applications. Genes (2023), 14, 483.
All of our services and products are intended for preclinical research use only and cannot be used to diagnose, treat or manage patients.