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Alpha-Mannosidosis (AM)

Animal models provide researchers with a means of understanding disease mechanisms, testing potential therapies, and assessing safety and efficacy. For alpha-mannosidosis, animal models are particularly important due to the rarity and complexity of the disease. Our company is committed to providing comprehensive disease modeling services and preclinical research services, including experimental design, animal model selection, data analysis, and report preparation. Our experts in rare disease drug development work closely with you and provide you with the tools and expertise you need to advance your research on drug discovery for alpha-mannosidosis.

Background

Alpha-mannosidosis is an autosomal recessive lysosomal storage disease (LSD) in which oligosaccharides accumulate due to a deficiency in the enzyme alpha-mannosidase. The disease affects multiple organs and systems, leading to a wide range of symptoms, including intellectual disability, skeletal abnormalities, hearing loss, and immune dysfunction. At present, there are no approved therapies for alpha-mannosidosis.

Animal models have played a crucial role in advancing researchers' understanding of the disease and developing potential therapies. Currently, mice, guinea pigs, and felines have been used as models for alpha-mannosidosis research, with each species providing unique insights into the disease. These animal models provide a valuable platform for the detailed study of pathology, oligosaccharide analysis, CNS cytopathology, and response to different therapies. This offers the possibility of developing therapeutic strategies such as enzyme replacement therapies (ERT) and gene therapies.

Our Services

We offer custom animal model generation services, allowing researchers to create animal models with specific genetic mutations or disease phenotypes. Our team of expert scientists and technicians work closely with our clients to ensure that their animal models are of the highest quality and meet their specific research needs. While none of the models can fully capture the complexity of human alpha-mannosidosis, each model mimics some aspect of the disease. Our animal models include, but are not limited to:

• Feline models
  We offer our clients feline models deficient in alpha-mannosidase activity and showing manifestations similar to human alpha-mannosidosis. The feline brain is well characterized functionally and physiologically, making these models useful in the study of central nervous system disorders such as alpha-mannosidosis.
• Mouse models
  We help our clients generate mouse models of α-mannosidosis by targeting the gene encoding lysosomal α-mannosidase. These mouse models can be used as proof-of-concept models for ERT.
• Guinea pig models
  We provide our clients with guinea pig models of α-mannosidosis with pathology very similar to that of human lysosomal storage disease. In addition to improving understanding of disease pathogenesis, these models also aid in the evaluation of therapeutic strategies.

Preclinical Research Services

Our animal models can be utilized for a range of preclinical research applications, including drug discovery, safety and toxicity testing, and biomarker identification. By using animal models, researchers can evaluate the safety and efficacy of potential therapies and obtain meaningful and promising data. These models can be used to test a variety of potential therapies, such as hematopoietic stem cell transplantation (HCT), ERT, and adeno-associated virus (AAV)-mediated gene therapy. Our preclinical platforms and services include, but are not limited to:

• Neurological examinations
• MANB enzymatic activity assay
• Oligosaccharide accumulation assay in the brain
• Alpha-mannosidase activity and morphology assay
• Immunohistochemistry
• Behavioral experiments

Our Advantages

• Practical experience with various animal disease models
• Excellent technical and experimental skills
• Gold-standard methods and novel molecular biology techniques
• Customized research solutions for your needs

We pride ourselves on our expertise and experience in animal modeling for rare genetic diseases like alpha-mannosidosis. Our animal models are carefully selected and bred to accurately reflect the genetic and phenotypic characteristics of the disease, ensuring that researchers have access to the most accurate and reliable models for their research needs. If you are interested in our disease modeling services, please contact us for more information.

Reference

• Yoon, S. Y.; et al. Clinical improvement of alpha-mannosidosis cat following a single cisterna magna infusion of AAV1. Molecular Therapy, 2016, 24(1): 26-33.

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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