Inducible HEK-293 cell line for TDP-43 aggregation studies

Technology
In development
University

The inducible HEK-293 cell line models TDP-43 aggregation without external stressors, enabling testing of small molecules and effectors for neurodegenerative disease research. It aids in evaluating their ability to inhibit aggregation and enhance autophagic pathways.

Overview

The inducible HEK-293 cell line is a novel model designed to study TDP-43 aggregation, a critical factor in neurodegenerative diseases. Unlike traditional methods that rely on stressors like arsenite or heat shock, this system induces aggregation through a highly aggregation-prone TDP-43 mutant, TDP-12XQ/N, in a controlled environment. This approach offers a more accurate representation of disease conditions and allows for the evaluation of therapeutic interventions aimed at restoring pre-mRNA processing functions.

Technical specifications

Key features:

  • Utilizes a genetically engineered HEK-293 cell line for inducible expression of TDP-43 mutant
  • Mimics loss-of-function effects and pre-mRNA processing alterations in a nuclear environment
  • Allows for high-throughput screening (HTS) of small molecules and cellular effectors
  • Enables study of autophagic and proteasomal pathways for aggregate removal
  • Compatible with proteomic and transcriptomic analyses for comprehensive understanding
Technology readiness level

This technology is currently at TRL 4, having been validated in a lab environment. It is set for further testing to assess its capabilities in inhibiting TDP-43 aggregation and facilitating aggregate removal, providing a promising platform for the development of neurodegenerative disease therapies.


About International Centre for Genetic Engineering and Biotechnology

The International Centre for Genetic Engineering and Biotechnology is an intergovernmental, non-profit research and training organization operating laboratories in Trieste, New Delhi, and Cape Town. Its campuses provide co-located labs and shared core facilities that enable collaborative R&D, while training and fellowship programs create sustained talent pipelines for partners. Proximity to innovation parks and leading universities at each site, plus biosafety and policy advisory services, helps industry navigate translation and regulatory contexts across regions. Research is supported by member-state contributions and competitive grants from national and international agencies. A dedicated technology transfer office manages IP, licensing, and commercialization across sites.

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