CRISPR Technology Rises to New Challenges

2022-09-29 Hits(91)

CRISPR excites ambition. Just consider a few CRISPR applications: gene drives (showing promise as a way to prevent malaria); ex vivo and in vivo gene therapies (entering clinical trials); and diagnostic tests (gaining momentum now that two assays have secured emergency use authorization for a raging pandemic disease—namely, COVID-19).

All of these applications can be traced back to research that was led by Emmanuelle Charpentier, PhD, and Jennifer Doudna, PhD. This research was introduced in a 2012 Science article that described the repurposing of a natural CRISPR system, an RNA-guided DNA endonuclease, as gene editing tool. Readers of the article may have noticed that its conclusion—that CRISPR "could offer considerable potential for gene targeting and genome editing applications"—was deliberately understated. Essentially, readers were invited to imagine how profound CRISPR applications might become, or how quickly these applications might leave the laboratory and enter the wider world.

Because CRISPR applications promise so many benefits, we are impatient to see them realized. Indeed, we may complain that the development of CRISPR therapies is too slow. Nonetheless, a handful of CRISPR therapies have advanced to the early stages of clinical trials, including therapies for sickle-cell anemia, HIV disease, and acute myeloid leukemia. And last year, a gene edited allogeneic chimeric antigen receptor T-cell therapy secured the FDA's regenerative medicine advanced therapy (RMAT) designation for the treatment of relapsed or refractory CD19-positive B-cell malignancies.

We are eager to see CRISPR succeed not just in medicine, but in other application areas where humanity faces serious challenges—areas that include crop production, bioenergy, manufacturing, and environmental remediation. To hasten progress in all these areas, scientists are working diligently to add tools to the CRISPR toolbox. A selection of the most interesting new tools are presented in this CRISPR anniversary article.

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