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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.

Schematic representation of mouse models in coloretal cancer. (Kucherlapati, M. H., 2023)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.

Why Choose Us?

Genetically Engineering Model Development Services

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.


  • 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.

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