CRISPR – A spotlight on gene therapy and its potential role in personalized medicine
August 01, 2017
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).
With such great potential for this technology, there come some daunting ethical concerns, and the scientific community is aware of and continually monitoring these, much like when cloning and stem cell research were first introduced. This technology may bring about a treatment for the most debilitating disorders, but could commercialization lead to the creation of designer-perfect babies? Who can define the line between what is considered normal versus a disability? As a preventative therapy, how do we determine what population is offered this opportunity? What role will the medical and legal systems play in determining the ethical monitoring of this powerful technology?
Truly, it’s a brave new world.
About the Author: Diana Trinh joined MD Buyline in April 2017 as a clinical analyst specializing in laboratory and pharmacy. She brought more than eight years of experience in medical laboratory science in several multi-disciplines, including microbiology, bone marrow biopsies, special coagulation, and flow cytometry at Parkland Health and Hospital System in Dallas. In addition, she served as a histocompatibility technologist in the ASHI-accredited Immunogenomics and Transplantation laboratory at Baylor University Medical Center in Dallas, North Texas’s largest transplant center. She also brings with her research laboratory experience in molecular and structural biology from UT Health San Antonio.
Trinh received her Bachelor of Science degree in Clinical Laboratory Science from UT Health San Antonio, and previously studied biology at Trinity University and at UT in San Antonio. She is a member of the American Society of Clinical Pathology and obtained her MLS(ASCP)CM certification in 2009.
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).
Diana Trinh
Truly, it’s a brave new world.
About the Author: Diana Trinh joined MD Buyline in April 2017 as a clinical analyst specializing in laboratory and pharmacy. She brought more than eight years of experience in medical laboratory science in several multi-disciplines, including microbiology, bone marrow biopsies, special coagulation, and flow cytometry at Parkland Health and Hospital System in Dallas. In addition, she served as a histocompatibility technologist in the ASHI-accredited Immunogenomics and Transplantation laboratory at Baylor University Medical Center in Dallas, North Texas’s largest transplant center. She also brings with her research laboratory experience in molecular and structural biology from UT Health San Antonio.
Trinh received her Bachelor of Science degree in Clinical Laboratory Science from UT Health San Antonio, and previously studied biology at Trinity University and at UT in San Antonio. She is a member of the American Society of Clinical Pathology and obtained her MLS(ASCP)CM certification in 2009.