Mucopolysaccharidosis Type IIIA (MPS IIIA)
Animal models play a critical role in advancing our understanding of lysosomal storage diseases (LSDs) and developing new therapies for these diseases. Our company offers a wide range of animal models and services to support mucopolysaccharidosis (MPS) research. Our models are highly reproducible, reliable, and correlated with human disease phenotypes, providing reliable data for preclinical studies. Our specialized animal modeling services will contribute to your research on the pathogenesis, development of therapies, and definition of biomarkers to assess therapeutic success.
Background
MPS disorders are a group of rare lysosomal diseases caused by genetic mutations that result in the loss of function of enzymes involved in glycosaminoglycan degradation. Incompletely degraded glycosaminoglycans accumulate in various tissues and organs, resulting in symptoms such as skeletal abnormalities, organ enlargement, and neurological deficits. There are currently 13 identified MPS medical types, including 2 discovered in the 2020s.
Animal models have provided valuable insights into glycosaminoglycan disease research. Mice, rats, dogs, and cats are among the animal species used as models for MPS and have proven particularly useful in the study of the disease. These animal models have been instrumental in advancing our understanding of the molecular and physiological consequences of MPS diseases and provide an opportunity to evaluate the effectiveness and safety of therapy interventions.
Fig. 1 Three cats with MPS show the typical features of this class of disease. (Haskins M E, 2007)
Our Services
Our team of scientists is dedicated to providing custom breeding services to generate animal models with specific genetic modifications and phenotypes to support our client's specific research needs. We work closely with our clients and provide expert guidance on animal model selection and study design. We offer models for different types of MPS, including MPS I, MPS II, MPS III, MPS IV, MPS VI, and MPS VII. Our disease modeling services include, but are not limited to:
- For type I MPS caused by α-L-iduronidase deficiency, we provide our clients with naturally occurring feline and canine congeners (including Plott hound dogs, Rottweiler dogs, and Boston terrier dogs) as reliable animal models. In addition, we specifically generate immunodeficient mouse models of type I MPS for our clients to evaluate human stem cell and gene therapies.
- For iduronate sulphatase deficiency (type II MPS), N-sulphoglucosamine sulphohydrolase (SGSH) deficiency (type IIIA MPS), and arylsulfatase B deficiency (type VI MPS), we offer animal models including mouse models (knockout mice or naturally occurring mice) or larger animal models (canine, cattle, goat, etc.) for preclinical therapeutic evaluation. We select the most appropriate animal model for our clients based on the strategy to be tested.
- For type IVA MPS without naturally occurring animal homologs, we help our clients engineer and generate immunotolerant mouse models that mimic the deficiency in N-acetylgalactosamine-6-sulphatase (GALNS).
- For type VII MPS with β-glucuronidase deficiency, we offer German shepherd dogs, GUS rats, and cats as preclinical research animal models.
Preclinical Research
Our MPS animal models exhibit significant pathogenic symptoms and lesions in the same organ system, and can be used to test the effectiveness and safety of various therapies, including:
- Enzyme replacement therapy (ERT)
- Bone marrow transplantation
- Gene therapy
- Substrate reduction therapy (SRT)
Our experts are dedicated to providing expert guidance and tailored solutions to support MPS research. In our company, we use state-of-the-art techniques to generate and maintain animal models, ensuring their health and welfare. Our animal models are available for purchase or can be accessed through our contract research services. If you are conducting preclinical research on MPS, please contact us for support.
Reference
- Haskins, M. E. Animal models for mucopolysaccharidosis disorders and their clinical relevance. Acta Pædiatrica, 2007, 96: 56-62.
All of our services and products are intended for preclinical research use only and cannot be used to diagnose, treat or manage patients.
