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Genome editing 'toolkit' to speed up gene therapy development

12 April 2021
Appeared in BioNews 1090

A genome editing consortium funded by the US National Institutes of Health (NIH) has unveiled plans to develop a new genome editing 'toolkit' to accelerate the development of gene therapies.

The Somatic Cell Genome Editing Consortium (SCGE) was granted $190 million to develop standardised genome editing approaches and methods, the outline for which was published last week. The overall goal is to facilitate the development of genome editing approaches for the treatment of multiple diseases, by providing a standardised starting platform. The consortium encompasses 75 principal investigators across 38 institutions.

'NIH realised it was important for all of us who are investigating gene editing to work together toward a common goal,' said author and SCGE member Professor Danith Ly of Carnegie Mellon University, Pennsylvania. 'What we'll end up with is a very valuable, rigorously evaluated resource for those who want to bring gene editing to patients,' he added.

An article published by SCGE members in Nature detailed the objectives of the grant. Over six years, 45 projects will focus on accelerating in vivo genome editing of human somatic cells, which the authors noted 'still faces substantial hurdles in terms of efficacy and safety'. Currently, genome editing has largely been limited to ex vivo settings, where cells are altered outside of the body before being reintroduced to the patient.

The article highlighted key focus areas for creating the 'SCGE Toolkit', including the development of novel technologies aside from CRISPR/Cas9 that can be used directly in vivo. Another major focus is to develop better delivery systems tailored for individual tissues, to allow selective editing that limits exposure to other parts of the body.

A core objective is the independent validation of genome editing technologies by a third-party, with the aim of increasing reproducibility of results across research groups – after three years, detailed methods of each new technology must be presented and tested at a separate facility before funding continues. Eventually, protocols will also be freely disseminated to the wider biomedical research community.

By standardising and expanding available genome editing technologies, the authors hope to reduce the time and cost of developing new therapies across an array of disease areas.

'Part of our efforts are intended to make these technologies accessible and scalable so you can get these potential treatments to large populations that have limited financial resources,' explained SCGE co-chair and joint senior author Professor Erik Sontheimer, of the University of Massachusetts.
 
He added: 'By working together on well-integrated projects, sharing ideas and swapping technologies in real-time, the goal is for the labs of the consortium to be more than the sum of their parts.'

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