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Project
Development of a neuromuscular system for testing therapeutic molecules in axonal Charcot-Marie-Tooth neuropathy.
We will study the formation and functioning of CMT2F and CMT2L patient-derived neuromuscular junctions, either by using monolayer co-cultures (microfluidic devices) or by using organoids. Both neuromuscular models will be used to characterize neuromuscular functionality/activity, and to investigate denervated or less well-structured neuromuscular junctions. The best applicable model will be used to test oligonucleotide and shRNA therapies for CMT2F and CMT2L caused by mutations in the small heat shock proteins HSPB1 and HSPB8, respectively.
Date:4 Oct 2022 → Today
Keywords:NEURO MUSCULAR DISEASES
Disciplines:Neurological and neuromuscular diseases