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SCGE Program Background
- Genome Editing allows precise corrections to be made in patients DNA and RNA
- CRISPR-Cas9 catalyzed development of experimental genomed editing therapeutics.
- Thousands of genetic diseases are amenable to targeted in vivo genome editing approaches
SCGE Phase 1
Gaps and Opportunities from 2017 Common Fund Planning Workshop
- Improved animal models to detect editing
- Human Cell Systems for measuring adverse events
- Delivery Systems for in vivo targeting
- Methods to track edited cells in vivo.
- Safer and more effective editors
SCGE Phase I addressed these gaps.
SCGE ToolkitAbout
The National Institutes of Health (NIH) has awarded over $140 million to help accelerate genome editing approaches from the lab to the clinic. The newly funded projects build upon the work performed though the Somatic Cell Genome Editing (SCGE) Program over the past five years and will focus on translating safe and effective genome editing therapeutics into the clinic.