Muscular Dystrophies Drug Discovery Service
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Muscular dystrophies refer to a group of inherited disorders characterized by progressive muscle weakness, wasting, and degeneration. Amongst these, Duchenne muscular dystrophy (DMD) is the most severe and common form. Currently, there is no effective treatment to stop the progression of the deadly disease, although several promising experimental strategies are currently being investigated.
Fig.1 The muscular dystrophies and organization of the dystrophin-glycoprotein complex. (Khurana, 2003)
Creative Biolabs' Molecular Mechanisms Research of Muscular Dystrophies
Genetically, MDs can be inherited in a dominant or recessive manner or, in many cases, caused by de novo mutations, which are therefore sporadic. Studies of muscle cells in muscular dystrophy have shown that mutated proteins cause perturbations of many cellular components. MDs have been associated with mutations in structural proteins, signaling molecules, and enzymes as well as mutations that result in aberrant processing of mRNA or alterations in post-translational modifications of proteins. These findings not only reveal important insights from cell biologists but also offer unexpected and exciting new approaches to therapy.
Creative Biolabs' Animal Models of Muscular Dystrophy
To explore therapeutic approaches, animal models are needed, and these should mimic human disorders. An obvious challenge in studying MDs is the heterogeneity of these diseases. This has led to the development of several animal models that are used experimentally to study some of the MDs. The most widely used experimental animal model of DMD is the mdx mouse. There are also models of muscular dystrophy in other species, such as dogs that lack dystrophin (CXMD dog) and the cardiomyopathic hamster, which lacks σ-sarcoglycan. Animal studies have used methods such as running, swimming, and electrical stimulation to determine the effects of training on animals with muscular dystrophy.
Present therapeutic strategies can be broadly divided into three groups: gene therapy approaches; cell therapy; and pharmacological therapy. Gene- and cell-based therapies offer the fundamental advantage of not having to correct secondary defects/pathologies (e.g., contractures) alone, especially if the treatment is started early in the disease. Gene therapy strategies attempt to deliver coding regions of the gene encoding dystrophin using specialized delivery vehicles (vectors). Cell-based strategies attempt to transplant muscle precursors such as myoblasts or stem cells taken from healthy donors, into dystrophin-deficient muscle.
Creative Biolabs' One-stop Shop Services for Muscular Dystrophy Preclinical Drug Discovery
Creative Biolabs prides itself on maintaining long-term relationships with its clients. By listening, discussing, and understanding your needs, we determine the solution that best fits your needs. As your partner, you can rely on our team of experts to assist in your disease research to advance your project. Please feel free to contact us for your Muscular Dystrophy research project.
- Khurana, T.S.; Davies, K.E. Pharmacological strategies for muscular dystrophy. Nature Reviews Drug Discovery. 2003, 2(5): 379-390.