CRISPR - What Is It, and How Can It Be Used In Medicine?
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This CME article delves into what CRISPR actually is, how it works, and what the ramifications are for disease treatment and society as a whole.
By Stuart M. Caplen, MD
The discovery of CRISPR, a gene editing technology, may turn out to be one of the most significant scientific advances in human history. We now have the ability to manipulate DNA relatively easily in ourselves and other animals, plants, and microscopic organisms. For the first time, humans can control their own genetic destiny. This article will delve into what CRISPR actually is, how it works, and what the ramifications are for disease treatment and society as a whole.
Jennifer Doudna, PhD and Emmanuelle Charpentier, PhD received the Nobel Prize in Chemistry in 2020 for their 2012 discovery of how to use CRISPR to edit the genome. Their work was truly groundbreaking and stood on the shoulders of other discoveries such as the structure of DNA by Watson and Crick, the Human Genomic Project which mapped out the complete human DNA, and previous researchers who discovered the CRISPR system and the nuclease enzyme Cas9 it uses to make cuts in genetic material. Before CRISPR it was possible to make changes to DNA, but it was cumbersome, time consuming, and expensive. CRISPR made redesigning genetic material a much faster and easier process.
What is CRISPR in Nature?
CRISPR is an acronym for Clustered Regularly Interspaced Short Palindromic Repeats. CRISPR is the process in nature by which a bacterial cell can identify, cut, and inactivate DNA sequences in viruses to protect itself from viral attacks. It was discovered by Francisco Mojica, PhD in 1993, who when sequencing bacterial DNA noticed that there were short segments of DNA that appeared to have no functionality, and were found at regular intervals. The segments were palindromic, meaning each side of the segment was a mirror image of the other side.
It was later discovered that these DNA segments……….
CRISPR Needs a PAM Sequence to Work
CRISPR Editing In the Laboratory
CRISPR Modifications ( Single Guide RNA, CRISPR Improvements, and Improved PAM Sequence Activation)
CRISPR Ethical Considerations
Medical Uses of CRISPR (Sickle Cell Disease and Thalassemia, Leber’s Congenital Amaurosis 10, and Other Potential Medical Uses)
Non-Medical Uses of CRISPR for Disease Control
Ecological Uses of CRISPR
and The Future
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ABOUT THE AUTHOR
Stuart M. Caplen, MD, FACEP, MSM
Dr. Caplen is a retired emergency medicine physician and former emergency department medical director, who also has a Master of Science in Management degree, and green belt certification in Lean/Six Sigma.
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