Induced Neurons for Study of Neurodegenerative Diseases
Research type
Research Study
Full title
Generation of induced neurons for in vitro and in vivo study of neurodegenerative disease and brain repair
IRAS ID
238160
Contact name
Roger Barker
Contact email
Sponsor organisation
Cambridge University Hospital NHS FT and University of Cambridge
Duration of Study in the UK
11 years, 9 months, 1 days
Research summary
We currently have no treatments that can alter the course of neurodegenerative diseases, such as Parkinson’s disease (PD), Huntington’s disease (HD), progressive supranuclear palsy (PSP), Multiple system atrophy (MSA), Amyotrophic Lateral Sclerosis (ALS), Multiple Sclerosis (MS), Gaucher disease (GD) and Alzheimer’s disease (AD). This is in part due to the lack of specific disease models that incorporate the problems that occur in patients. In this study we plan to use a new model involving patient cells to study the mechanisms that cause this group of conditions, so that we can identify targets for treatment. This model is based on the use of induced neurons (iNs) which are generated from patient skin cells (fibroblasts).
Skin biopsies have been obtained from patients with neurodegenerative disease and healthy controls as part of one of the linked project (REC09/H0311/88). These have been cultured to provide a supply of fibroblasts. These fibroblasts will be converted into stem cells, iNs, and induced glial cells (iGs). This proposed project involves using the reprogrammed neural cells to study the mechanisms of aging as well as those that lead to the aforementioned neurodegenerative diseases. This will be performed by analysing these neurons for genetic and biochemical abnormalities in culture dishes, and also after grafting them into rats. The reprogrammed cells will be used to test whether potential treatments are able to correct the problems that cause neurodegenerative diseases - if they are able to do this in this model they will be good candidates to take into clinical trials in patients.
Additionally, it may be possible to use these reprogrammed cells to replace the nerve cells that are lost in neurodegenerative diseases, as a cell-based therapy. Part of this study will also therefore involve grafting the neural cells into rat models of these diseases, to see if they can improve their deficits.
REC name
London - Central Research Ethics Committee
REC reference
18/LO/0615
Date of REC Opinion
6 Apr 2018
REC opinion
Favourable Opinion