June 1, 2018 (Vol. 38, No. 11)

Moving Gene Editing from Basic Research to the Clinic

You know biotechnology is capturing the public’s imagination when it becomes the premise of a Hollywood blockbuster. In “Rampage,” one of the big movie releases this spring, Dwayne “The Rock” Johnson saves mankind from the havoc wreaked by a trio of giant mutant animals produced by an accidental misapplication of CRISPR technology.

It is hardly a spoiler to divulge that even the latest and greatest form of gene editing is no match for The Rock. An action hero is about the only thing that could stop CRISPR in its tracks. As the contents of this supplement reveal, those tracks are beating a clear path from basic research to more clinical applications. Several biotech and pharma companies are close to applying for regulatory approval to launch CRISPR-based clinical trials in the United States and Europe for a range of conditions, including sickle-cell anemia, hemophilia, and rare hereditary forms of blindness.

The five articles in this supplement cover a range of new CRISPR-based applications, including properties of new nucleases beyond the original Cas9. In “CRISPR Shakes Up Tools and Tech”, Christina Bennett reviews highlights from the recent Precision CRISPR Congress. For example, she recalls how presenter Janice Chen described new diagnostic applications of CRISPR from Jennifer Doudna’s lab at UC Berkeley, some of which are being commercialized in a new company called Mammoth Biosciences.

Further RNA-targeting applications are considered by Richard Stein in “The Expanding CRISPR-Cas Toolbox”, and Caroline Seydel discusses an array of genome editing innovations that are compatible with Cas9 nucleases in “Pushing the Boundaries of CRISPR”.

As the arc of this supplement makes clear, CRISPR is showing tremendous medical potential. In “CRISPR Enters the Clinic”, Josh Roberts details progress by CRISPR Therapeutics, Juno Therapeutics, and academic groups in honing CRISPR gene editing for clinical applications. The Q&A with law professor Jacob Sherkow, in “CRISPR in the Courthouse”, is based on a podcast that appears on The CRISPR Journal website. Rounding out the supplement is a tutorial “Analyzing CRISPR Editing Results“, produced by Synthego, on a new freely available tool called ICE (Inference of CRISPR Edits).

For further information on CRISPR, stay tuned to GEN and check out The CRISPR Journal—the new peer-reviewed journal on all aspects of genome editing research.

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