Guidelines for pre-clinical animal and cellular models of MuSK-myasthenia gravis

摘要: Muscle-specific tyrosine kinase (MuSK) autoantibodies are the hallmark of a form myasthenia gravis (MG) that can challenge neurologist and experimentalist. The clinical disease be difficult to treat effectively. MuSK affect neuromuscular junction in several ways. When added muscle cells culture, antibodies disperse acetylcholine receptor clusters. Experimental animals actively immunized with develop weakness. Weakness is associated reduced postsynaptic numbers, amplitudes miniature endplate potentials potentials, failure transmission. Similar impairments have been found mice injected IgG from MG patients positive for autoantibody (MuSK-MG). active passive models begun reveal mechanisms by which disrupt synaptic function at junction, should valuable developing therapies MuSK-MG. However, translation into new improved treatments requires procedures not too cumbersome but suitable examining different aspects effects potential therapies. Study design, conduct analysis carefully considered transparently reported. Here we review what has learnt animal culture MuSK-MG, offer guidelines experimental design studies, including sample size determination, randomization, outcome parameters precautions objective data analysis. These principles may also relevant increasing number other antibody-mediated diseases now recognized.