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Generation of ultra-deep libraries of transcriptional activators for gene therapy

Project description

A modular system for the production of transcriptional activators

Human gene therapy entails the administration of genetic material to alter the expression of or modify endogenous genes. In cases where activation of gene expression is required, researchers transfer transcription factors – proteins necessary for gene transcription – to the target cells. The EU-funded Diverge project has developed a modular system that combines site-specific recombinase and molecular splicing to generate billions of transcription factor variants in vivo from a single construct. Researchers are exploring the potential to commercialise this system and expand the applicability of the technology in therapy and biotechnology.

Objective

We have generated a novel genetic circuit that, by combining an unusual site-specific recombinase and molecular splicing, allows to functionally diversify modular proteins in-vivo in a highly-directed manner. Applying this method to modular transcriptional activators allowed us to generate billions and even trillions of transcription factors variants in-vivo starting from a single base construct. The aim of the DIVERGE PoC project is to to explore the commercial potential of this approach for the isolation of transcriptional activators for human gene activation therapy and to establish a strategy for commercialisation of current and conceivable future implementations of this technology.

Host institution

IMPERIAL COLLEGE OF SCIENCE TECHNOLOGY AND MEDICINE
Net EU contribution
€ 150 000,00
Address
SOUTH KENSINGTON CAMPUS EXHIBITION ROAD
SW7 2AZ LONDON
United Kingdom

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Region
London Inner London — West Camden and City of London
Activity type
Higher or Secondary Education Establishments
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Total cost
€ 150 000,00

Beneficiaries (1)