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<span>Somatic Cell Genome Editing</span>
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<h1>Somatic Cell Genome Editing</h1>
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Genome editing is a promising technology that could offer new treatments and cures for diseases. We are leading the NIH-wide working group to manage the SCGE program.
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<h2 class="section-heading">About Somatic Cell Genome Editing</h2>
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</style><div class="contact-box"><p><strong>Contact</strong> <br><a href="mailto:pj.brooks@nih.gov"><u>P.J. Brooks, Ph.D.</u></a></p></div><p>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.</p><p>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 many genetic diseases.</p><p>In January 2018, <a href="https://www.nih.gov/news-events/news-releases/nih-launch-genome-editing-research-program">the NIH Common Fund launched a new program</a> focusing on somatic cell research. The first phase of the <a href="https://commonfund.nih.gov/editing">Somatic Cell Genome Editing (SCGE) program</a> created high-quality tools for safe and effective genome editing in humans and made these tools widely available to the research community to reduce the time and cost of creating new therapies.</p><p>We led the NIH-wide working group composed of staff from several other NIH institutes and centers. The program was organized around these aims:</p><ul><li>Expand the number of genome editing tools available</li><li>Create delivery systems that can efficiently target the cells of specific organs and tissues in the human body</li><li>Design new assays for testing the safety and effectiveness of genome editing and delivery tools</li><li>Share the knowledge, methods and tools developed through this program with the scientific community through the <a href="https://scge.mcw.edu/toolkit/">SCGE Toolkit</a></li></ul><p>Building on the success of Phase 1, the next phase of the SCGE program launched in 2023. The goal of Phase 2 is to speed the translation of genome editing therapies into the clinic. Along with the National Institute of Neurological Disorders and Stroke, we led another NIH-wide working group to:</p><ul><li>Advance the evaluation and clinical development of novel genome editing therapies</li><li>Create regulatory pathways to the clinic for multiple diseases at a time</li><li>Share successful strategies to start <em>in vivo</em> genome editing studies in humans</li><li>Develop targeted delivery technologies through the <a href="https://www.freelancer.com/nih/targeted-challenge">TARGETED Challenge</a></li></ul><p><a href="https://commonfund.nih.gov/editing">Learn More About SCGE</a>.</p>
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<h2>Genome Editing to “Re-Write” Wrongs</h2>
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<p>Learn more about the NIH Common Fund's Somatic Cell Genome Editing program in this <em>Nature Reviews Drug Discovery </em>article.</p>
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