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CRISPR – A spotlight on gene therapy and its potential role in personalized medicine

August 01, 2017
Business Affairs Pathology
By Diana Trinh, MLS(ASCP)CM
Clinical Analyst, MD Buyline


Scientists, professors, laboratory professionals, and vendors from a wide array of diagnostic specialties are gathering at the 69th American Association of Clinical Chemistry (AACC) Annual Scientific Meeting & Clinical Lab Expo in San Diego this week.

They’ll be presenting topics relating to ways of tackling current issues and showcasing innovations in laboratory automation, data analytics, and diagnostics. Diana Trinh and Dennis Matricardi, clinical analysts from MD Buyline, will be filing reports exclusive to HealthCareBusiness News from the meetings and expo.



In laboratory medicine, the focus is usually on diagnostics, but a new research field has developed in the laboratory in molecular-based therapeutics.

AACC invited Jennifer Doudna, professor of molecular and cellular biology at UC Berkley, to deliver the keynote speech, in which she addressed her work in CRISPR-mediated genome editing. Doudna has been featured in many scientific journals as well as several popular magazines such as Wired and Time magazine.

DNA is the widely recognizable double helix molecule that stores the genetic instructions for all living things. In the realm of molecular diagnostics, great strides have been made in pinpointing genetic diseases and cancers, as well as detecting some polymorphisms that make us respond differently to certain medications. Now that we have deciphered some of the DNA code, it might be possible to actually change that code.

With the introduction of CRISPR (clustered regularly interspaced short palindromic repeats) technology, DNA can now be precisely and efficiently cut and edited like the Search and Replace functions in a Word document. This technology was actually discovered in bacteria as a defense mechanism against viruses. When a virus injects its DNA into a bacterium, the CRISPR complex comes into play to cut and destroy. Doudna and her team were able to extract this bacterial mechanism and enhance it to target not only viruses, but any DNA sequence. These complexes can be programmed with guide RNA sequences to search and cut, then new genetic code can be inserted.

Currently, there are no patient clinical trials in the U.S. involving this technology. But several researchers have utilized this CRISPR technology on human cells and on animal models such as tadpoles, monkeys, and mice, demonstrating the efficacy of this technology. Some of Doudna’s current research focuses on gene editing for brain disorders such as Parkinson’s, Huntington’s, and Amyotrophic Lateral Sclerosis (ALS).

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