The availability of animal models that mimic human diseases has greatly increased our understanding of neuromuscular rare disease pathology. Our company is committed to providing our clients with a wide range of animal models, including not only naturally occurring animal models but also custom models carrying specific mutations. Our advances in gene editing allow us to help our clients develop more accurate disease models, which greatly expands the availability of clinically relevant models. With our well-designed and reliable preclinical studies that use predictable models, the development of new therapies for rare neuromuscular diseases is expected to make new progress.

Background

Neuromuscular diseases are a heterogeneous group of rare inherited disorders that impair peripheral nerve and/or muscle function and eventually lead to muscle weakness, wasting, and other complications. Neuromuscular diseases include a wide range of rare disorders, including amyotrophic lateral sclerosis (ALS), Huntington's disease, neuromuscular junction disorders myasthenia gravis, and more. With limited treatment options available, there is a growing need to design effective preclinical studies to achieve greater success in clinical trials of new therapies.

Recent advances in genetics and molecular biology have led to the development of animal models that mimic the symptoms of these diseases. These models provide valuable insight into their underlying mechanisms. Although animal models do not fully recapitulate human pathology and there are differences in disease severity between them, they are essential for assessing drug safety and efficacy and for helping prioritize compounds for further clinical development.

Fig. 1 Examples of four mice models for neuromuscular diseases. (Vainzof M, et al., 2008)

Neuromuscular Rare Diseases Modeling Services

We specialize in generating genetically modified animal models that recapitulate neuromuscular rare disease symptoms and pathology. We offer a range of services, including gene editing, transgenic animal production, and phenotypic analysis, to help researchers investigate the mechanisms of these diseases and develop new therapies.

Our extensive animal modeling services cover multiple species. We offer small animal models such as zebrafish, Drosophila, and C. elegans, which are advantageous for high-throughput drug screening and mechanistic and functional studies. We also offer larger animal models, including rodent, dog, pig, and cat models, which better recapitulate the various facets of human diseases. Our services include but are not limited to:

Animal Models for ALS Disease

Animal Models for Huntington's Disease

Animal Models of Neuromuscular Junction Disorders

Animal Models for Sarcoglycanopathy

Animal Models for Duchenne Muscular Dystrophy

Animal Models for Calpainopathy

In addition to generating animal models, we also offer comprehensive phenotypic analysis services to help researchers characterize the disease phenotype and monitor disease progression. Our services include histological analysis, electromyography (EMG), behavioral tests, and other techniques that provide valuable insights into disease pathology.