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Clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 is a genome-editing technology  1 , 2   that utilizes archaeal and bacterial Cas9 nucleases to introduce double-stranded breaks in DNA at targeted sites. These breaks can be used to remove, replace, or add pieces of DNA. While not the first genome editor, CRISPR-Cas9 is efficient and cost-effective because cutting is guided by a strand of RNA rather than a protein. The potential uses in health care are plentiful, from disrupting dominant genes that cause cancer  3   to repairing mutated genes that cause genetic diseases, such as muscular dystrophy  4  . Therapeutic approaches based on this technology fill the preclinical pipeline, and rely on the use of viral vectors to deliver the  Cas9  gene and guide RNA to a gene of interest. However, concerns regarding the safety and efficacy of CRISPR-Cas9 use in gene therapy remain. A pre-print released prior to peer review has recently underlined the question of whether immunological responses to Cas9 may negatively impact its clinical use  5  . Here we discuss the implications of this finding for the application of CRISPR/Cas in gene therapy.

Cas9 immunity creates challenges for CRISPR gene editing therapies