Identifying and characterizing new therapy targets in TDP-43 proteinopathies

Summary

This project aims to identify and characterize new therapeutic targets for TDP-43 proteinopathies, a group of neurodegenerative diseases including ALS and FTD, by investigating the role of ATXN3 alternative polyadenylation and identifying novel TDP-43 target genes in human neurons.

What they want

The project will determine the functional significance of ATXN3 alternative polyadenylation (APA) and identify cellular pathways to modulate ATXN3 expression in human neurons. It will also identify and characterize new TDP-43 target genes in human neurons, specifically by studying the function of MARK3 APA and by developing a new cellular tool to precisely define the transcriptome of human neurons undergoing TDP-43 nuclear clearance in a cell-type and temporally-controllable manner.

Deliverables

Functional significance of ATXN3 alternative polyadenylation determined
Cellular pathways to modulate ATXN3 expression in human neurons identified
New TDP-43 target genes in human neurons identified and characterized
Function of MARK3 APA studied
New cellular tool developed to define the transcriptome of human neurons undergoing TDP-43 nuclear clearance

Technical requirements

Use of human iPSC-derived neurons
Analysis of ALS/FTD patient brain tissue
Techniques for studying alternative polyadenylation (APA)
Transcriptome analysis
Development of cellular tools for cell-type and temporally-controllable studies

Market context

inferred from NAICS

Professional, Scientific & Technical Services

NAICS 541714

US market size: $2.0T
Typical award: $25K – $50M

Typical buyers

All federal civilianDoDStates

Commonly required

8(a)WOSBSDVOSBPE/PMP

Sector-level estimate — full code lookup not yet in catalog.