CRISPR and Gene Editing
In 2015, the advantages of the clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-associated protein (Cas)9-mediated genome modification enabled us to edit the genomes of a variety of organisms rapidly and efficiently for biological and therapeutic applications. (6)
Cas proteins are endonucleases that use a single guide RNA (sgRNA) to form complementary base pairs with target DNA and then cleave the DNA at specific sites. Among the different types of Cas proteins, Cas9 is the most widely used type because of its simplicity, high efficacy, and ease to use. One major concern about this system is its specificity, which has changed the field of gene editing by making it much simpler and faster to modify DNA sequences with high precision. (7)
In 2016, a Chinese group initiated the first clinical study of CRISPR-Cas9 by injecting Cas9-engineered T cells to a patient with metastatic non-small cell lung cancer (NSCLC). (8) The result was promising. Another trial with a similar approach is currently running in the US. In addition, CRISPR technology could also be used to improve cancer therapies such as CAR-T.
However, the CRISPR-Cas9 system is still in its early stages. Similar as gene therapy, delivery of the CRISPR-Cas9 system to target tissues or cells in human body is the biggest challenge for its therapeutic applications. Development of safe and efficient delivery systems is therefore crucial for the success of CRISPR-Cas9 in humans.
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