Somatic Cell Genome Editing

Illustration of DNA modification

Illustration of DNA modification (Shutterstock, vchal)

Genome editing is a promising technology that could offer new treatments and cures for diseases. The human genome has thousands of genes, each of which has the information that cells use to make protein. The human body needs a vast number of specific proteins to work properly. In genetic diseases, a gene has a problem that means a protein is made wrong — or not made at all. Genome editing changes the DNA sequence so that the cells can correctly make proteins again. Progress in genome editing could help treat thousands of genetic diseases.

The Importance of Somatic Cells

In the past decade, researchers have advanced genome editing technology to allow precise changes to the DNA code inside live cells. Using this technology, scientists could edit disease-causing DNA within the body’s non-reproductive cells, known as somatic cells. Making this technology more efficient and limiting the edits to these cells lowers the risk of having unintended editing-related changes passed down to future generations. Researchers could use this technology to potentially treat a large number of genetic diseases.

Expanding Genome Editing Research

In January 2018, recognizing the impact of this research, the NIH Common Fund launched a new program focusing on somatic cell research. The first phase of the Somatic Cell Genome Editing (SCGE) program aims to create high-quality tools for safe and effective genome editing in humans and make these tools widely available to the research community to reduce the time and cost of creating new therapies.

NCATS led the trans-NIH working group composed of staff from several other NIH institutes and centers that managed the new program. The joint program aims to:

  • Expand the number of genome editing tools available.
  • Create delivery systems that can efficiently target the cells of specific organs and tissues in the human body.
  • Design new assays for testing the safety and effectiveness of genome editing and delivery tools.
  • Share the knowledge, methods and tools developed through this program with the scientific community through the SCGE Toolkit.

Building on the success of Phase 1, the next phase of the SCGE program launches in 2023. The goal of Phase 2 is to speed the translation of genome editing therapies into the clinic. NCATS and the National Institute of Neurological Disorders and Stroke will lead another trans-NIH working group to:

  • Advance the evaluation and clinical development of novel genome editing therapies.
  • Create regulatory pathways to the clinic for multiple diseases at a time.
  • Share successful strategies to start in vivo genome editing studies in humans.
  • Develop targeted delivery technologies through the TARGETED Challenge.

To learn more about the SCGE program, visit the NIH Common Fund website

Genome Editing to 'Re-Write' Wrongs

Learn more about the NIH Common Fund's Somatic Cell Genome Editing program in this Nature Reviews Drug Discovery article.

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P.J. Brooks, Ph.D.