Human Genome Editing FAQ

This FAQ document focuses on the utilization of genome editing in humans to treat disease. It answers basic questions such as “What is genome editing and how does it work?” to more detailed questions, such as “What are the oversight and regulatory frameworks for approval of somatic genome editing therapies”.

One takeaway from this document is that all current therapeutic R&D intended for FDA approval involves “somatic” cells (skin, muscle, lung, liver, heart, or blood cells), not “germline” cells (sperm or egg). This is an important distinction and is described in the FAQ.

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