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The NIH Somatic Cell Genome Editing program

In a recent paper published in Nature, the National Institutes of Health (NIH) discusses the Somatic Cell Genome Editing Consortium and their plans to develop and benchmark gene editing approaches.

Genome editing

Genetic risk factors are important in human disease. They can be inherited, infectious and malignant. Researchers for decades have hoped to target these factors by modifying the genomes of patients to correct the mutation, disable pathogen genomes, arm immune cells to attack tumours and develop new therapeutic opportunities. Gene therapy is an example of where gene addition has shown successful therapeutic value. However, in many other cases, the genome of the patient must be edited in order to achieve therapeutic benefit.

Genome editing encompasses a range of diverse technologies that can make different genomic alterations in different contexts. The most prominent technique is CRISPR/Cas9 which has revolutionised the field of genome editing. Nonetheless, no platform is appropriate for all contexts. Several factors are critical to editing success including efficiency, precision and accuracy.

The Somatic Cell Genome Editing Consortium

The Somatic Cell Genome Editing (SCGE) Consortium has been established by the NIH to accelerate the development of safer and more-effective methods to edit the genomes of disease-relevant somatic cells in patients. The NIH has allocated around $190 million over 6 years in support of the SCGE Consortium. The Consortium now includes 72 principal investigators from 38 institutions that are pursuing 45 distinct but well-integrated projects.

One of the key challenges within the field is the comparison of various technologies. Therefore, the Consortium aims to develop paths that will enable the mixing and matching of various technologies. In addition, a key value of the Consortium is transparency. The team will enable others to access its research output and use its results and products to inform and accelerate their own disease-focused projects. Overall, the end goal of the Consortium is to produce a freely available toolkit that will provide the research community with rigorously evaluated information regarding genome editors and methods for delivering and tracking gene editing molecules.

The Consortium are strictly focussed on somatic editing. They note in the paper that germline editing is excluded as a goal and should be considered as an unacceptable outcome that should be carefully prevented. Moreover, while much of their work focuses on CRISPR-Cas related systems, the SCGE points out that it is also important to continue to develop other systems. They specifically describe peptide nucleic acid-based gene editing. This technique is administered intravenously and edit cells in vivo. Peptide nucleic acids (PNAs) are relatively small, synthetic molecules that recognise specific DNA sequences. While they do not produce the same yield as CRISPR-Cas systems, they are less likely to make off-target modifications.

Overall, the SCGE program is designed to advance the field of genome editing towards a broader spectrum of human therapeutic applications.

Image credit: By FatCamera –

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Base Editing / CRISPR / Gene Editing

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