21 Jun Rapid Improvements Coming to Gene Editing Techniques
MedicalResearch.com Interview with:
Michael Farzan PhD
Co-chair and Professor
Department of Immunology and Microbiology
The Scripps Research Institute
MedicalResearch.com: What is the background for this study? What are the main findings?
Response: CRISPR is system for immune protection of bacteria. It has now been widely adopted for use in editing mammalian cells. The most commonly used CRISPR effector protein is Cas9. Cas9 binds a guide RNA to recognize a DNA target, for example an incoming virus infecting a bacterium, or a gene in a human chromosome. In bacteria, Cas9 requires a second protein to clear the guide RNA from a longer “CRISPR array”, basically a string of guide RNAs.
We have been studying a CRISPR effector protein related to Cas9 called Cpf1. In bacteria it was know that, unlike Cas9, Cpf1 could cleave a CRISPR array by itself, without assistance from a second protein. We knew that if it could do the same thing in human cells, it would help to simplify a number of gene-editing applications. We were able to show that Cas9 could indeed excise multiple guide RNAs from a single message RNA in human cells. We further showed that this approach was more efficient than the previous ways that guide RNAs were generated for gene editing, even more so when multiple guide RNAs were needed.
MedicalResearch.com: What should clinicians and patients take away from your report?
Response: Mostly that improvements to gene editing techniques are coming at a very fast pace, and these improvements will change the way we treat a number of disorders, including muscular dystrophy, cystic fibrosis, hepatitis B infection, and I am sure many, many others.
MedicalResearch.com: What recommendations do you have for future research as a result of this study?
Response: Our study adds to the growing list of reasons that Cpf1 may be more useful for many gene editing applications than Cas9. It suggests that the field should focus its attention on the further optimization of Cpf1, and the further characterization of other Cpf1 orthologs.
MedicalResearch.com: Is there anything else you would like to add?
Response: Guocai Zhong was the major intellectual contributor to this study, which is why – unusually for a first author – he serves as corresponding author on the paper. We don’t have any disclosures.
MedicalResearch.com: Thank you for your contribution to the MedicalResearch.com community.
Note: Content is Not intended as medical advice. Please consult your health care provider regarding your specific medical condition and questions.