Transcriptional profiling of cell types vulnerable to degeneration
Research type
Research Study
Full title
Transcriptional profiling of defined, vulnerable cell types associated with human psychiatric disorders and neurodegenerative diseases for the identification of novel targets for drug discovery.
IRAS ID
225921
Contact name
Mark Carlton
Contact email
Sponsor organisation
Cerevance Ltd.
Duration of Study in the UK
0 years, 10 months, 0 days
Research summary
Transcriptional profiling of defined, vulnerable cell types associated with human psychiatric disorders and neurodegenerative diseases for the identification of novel targets for drug discovery.
Cerevance is a pharmaceutical company focused on developing treatments for central nervous system (CNS) disorders. Using a powerful technology platform termed NETSseq (Nuclear Enriched Transcript Sort sequencing) we aim to examine the molecular diversity of individual neuronal cell types in human tissue in order to understand their genetic complexity, the contribution that each of these make to circuit function and, in the context of brain disorders, their selective vulnerability.
The NETSseq technology represents the next evolution of transcriptomics analysis that is amenable to human post-mortem brain tissue. From UK tissue banks we will request snap frozen donor brain tissue to build a control dataset of transcriptomes from cell types selected for relevance to human disease covering both sexes and spanning different ages. This data will be compared to transcriptomes generated from donor samples obtained from diseased populations likely to include Huntington’s, Parkinson’s, Alzheimer’s, Motor Neurone Disease and potentially ataxias.
The datasets generated will undergo bioinformatic analysis to identify genes showing unique expression patterns in identified cell types. It is hoped that the data will provide novel insights and identify gene hubs or pathways with potential importance for studying human disease pathophysiology and suggest new targets for drug discovery to aid the generation of new medicines towards these devastating and often poorly treated CNS diseases.
The implementation of this new transcriptomics technology platform will deliver human expression data relevant to human brain disorders on a scale previously unachievable. Such data will produce novel insights into disease pathology and biological phenomena that could not be detected using existing standard bulk genomic or transcriptomics interrogations and is capable of a throughput not possible with current single-cell sequencing based technologies.REC name
East Midlands - Derby Research Ethics Committee
REC reference
17/EM/0173
Date of REC Opinion
4 May 2017
REC opinion
Favourable Opinion