Transcriptional profiling of vulnerable cell types in disease
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
257099
Contact name
Mark Carlton
Contact email
Sponsor organisation
Cerevance Ltd.
Duration of Study in the UK
2 years, 0 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.\n\nCerevance 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 are examining 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. \nThe NETSseq technology represents a progressive evolution of existing transcriptomics methodology that is amenable to human post-mortem brain tissue. Our current study hopes to build on our early validation work and the insights we have already made into human disease and expand the scope of our analysis into additional diseases including both neurodegenerative conditions and psychiatric diseases.\nAs with our previous project the aim is to produce disease-relevant, cell specific transcriptional fingerprints. The datasets generated will undergo bioinformatic analysis to identify genes showing unique expression patterns in identified cell types. These data have already been used to seed an internal research pipeline based on novel findings that identify gene hubs or pathways that are implicated in the disease process of neurodegenerative diseases. It is the overall aim that this research will assist the generation of more effective therapies against these devastating and often poorly treated CNS diseases.\nExpanding our transcriptomics technology platform will deliver further expression data relevant to human brain disorders on a scale previously unachievable. The data are proving to be useful in the characterisation of disease pathology and biological phenomena that could not be detected using existing standard bulk genomic or transcriptomics interrogations.\n
REC name
South West - Cornwall & Plymouth Research Ethics Committee
REC reference
18/SW/0273
Date of REC Opinion
26 Nov 2018
REC opinion
Favourable Opinion