Development of a new human in vitro model to study neuromuscular diseases
The complex processes of initiation and coordination of signaling between the central nervous system (CNS) and muscles are highly susceptible to damage in neuromuscular disease (NMD). This vulnerability is positioned most prominently at the neuromuscular junction (NMJ), where motor neurons generate muscle contraction by releasing acetylcholine into the synaptic cleft. Sadly, partially due to a lack of complete scientific understanding of the human NMJ, there is currently no cure for NMDs characterized by dysfunctional NMJs. While the cellular and molecular features of mouse NMJs have been characterized in depth, there is much less known about the human NMJ. Thus, it is not surprising that drugs found to alleviate symptoms in NMD animal models by acting at the NMJ have frequently failed in clinical trials. In order to expand our understanding in ways that will lead to effective treatments for patients, the field needs to move beyond animal models and directly study human NMJs. I propose to address this unmet need by modeling NMD-related NMJ dysfunction with human cells from patients. I hypothesize that this approach will identify novel targets and pathways, thereby establishing a platform for the discovery of drugs that will effectively treat human NMDs.
Date: 3 November 2023, 15:00
Venue: IMS-Tetsuya Nakamura Building, Roosevelt Dr, Headington OX3 7TY
Venue Details: IDRM Seminar Rooms
Speaker: Assistant Professor Helen Miranda (Case Western Reserve University ​)
Organisers: Dr Thomas Roberts (Department of Paediatrics), Dr Rebecca Berrens (Department of Paediatrics), Dr Filipa Simões (DPAG, IDRM, University of Oxford), Associate Professor Carlo Rinaldi (University of Oxford, MDUK Oxford Neuromuscular Centre), Dr Oliver Stone (University of Oxford, Department of Physiology, Anatomy and Genetics (DPAG))
Organiser contact email address: idrmoperations-pa@idrm.ox.ac.uk
Host: Prof. Carlo Rinaldi (University of Oxford)
Part of: IDRM Seminar Series
Booking required?: Required
Booking email: idrmoperations-pa@idrm.ox.ac.uk
Audience: Members of the University only
Editor: Rufina Kaloyanova