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JAX Source

The Jackson Laboratory


This resource manual summarizes The Jackson Laboratory's extensive resources for SMA (Spinal Muscular Atrophy) research. Its purpose is to guide you through your selection of the best model for your studies. For accurate interpretation of results, it is essential to understand the differences that exist in the spectrum of disease as it is modeled by a mouse - not only on a phenotypic level but on the molecular defect level as well, and recognize that not all phenotypes displayed by mouse models may be relevant in the clinic. Nonetheless, mouse models have proven essential in both basic research and preclinical discovery for bringing promising therapeutics to the clinic for SMA patients.