Overview
Are microvascular defects relevant in Spinal Muscular Atrophy?: Characterisation of the mouse model
- Principal investigator(s):
- Prof. Dr. Simon Parson
- Institution:
- University of Aberdeen, UK
- Grant:
- €98,110
- Grant Type:
- Operating Grant
- Start Year:
- 2024
- Duration:
- 1 year
- Call number:
- 12
- Status:
- Ongoing
Prof. Dr. Simon Parson
Prof. Dr. Parson is Regius Professor of Anatomy at the University of Aberdeen in the UK. He was educated at Durham University (BSc Zoology, 1987) and the University of Edinburgh (PhD Neuroscience, 1990) and was President of the Anatomical Society between 2019-2022.
In Focus
Background
SMA is caused by a decrease in an essential protein called SMN, present in all cells and required for cell survival. While the pathology in nerves and muscles is most significant in people living with SMA, Prof. Parson and his team have shown wide ranging changes in the cardiovascular, gastrointestinal and renal systems, and observed an underlying defect in the blood vessels which supply these systems. Importantly, defects in blood vessels are associated with nerve cell loss.
Building on a 1-year SMA Europe operational grant obtained through the 11th Call for Research proposals, with this grant Prof. Parson and his team will set out to determine if these defects in blood vessels may cause or worsen patient symptoms.
How will Prof. Dr. Parson’s team do this?
During the period funded by the 11th Call for Research proposals, Prof. Parson and his team have generated a new transgenic mouse that specifically lacks the SMN protein in the blood vessels. The team will now characterize this model, to better understand the implications of vascular pathology in SMA.
Why is it interesting to patients?
Studying vascular pathology in SMA will help guide the development of novel therapies targeting blood vessels and associated pathologies, with the goal of improving outcomes for individuals living with SMA.