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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.[1] 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.[2] 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[1], 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……….

More about:

  • 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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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.


[1] CRISPR Timeline. Broad Institute. 2021. Retrieved from:

[2] CRISPR Explained. Novateinbio. 2019.

[3] Importance of the PAM Sequence in CRISPR Experiments. Synthego. 2021. Retrieved from:

[4] The Complete Guide to Understanding CRISPR sgRNA. Synthego. 2021 Retrieved from:

[5] Isaacson W, The Codebreaker. Simon & Schuster. March 9, 2021.

[6] Crossley M. What is CRISPR gene editing, and how does it work? The conversation. January 31, 2018. Retrieved from:

[7] Super-precise new CRISPR tool could tackle a plethora of genetic diseases. Nature. 21 October 2019. Retrieved from:

[8] New kind of CRISPR technology to target RNA, including RNA viruses like coronavirus. Science Daily. March 16, 2020. Retrieved from:

[9] Integrated DNA Technologies. 2021. Retrieved from:

[10] McDade J. The PAM Requirement and Expanding CRISPR Beyond SpCas9. Addgene. Updated Aug 20, 2020. Retrieved from:

[11] Cyranoski D. The CRISPR-baby scandal: what’s next for human gene-editing. Nature. 26 February 2019. Retrieved from:

[12] Wee SL. Chinese Scientist Who Genetically Edited Babies Gets 3 Years in Prison. The New York Times. Dec. 30, 2019. Retrieved from:

[13] Michael Eisenstein. Gene therapies close in on a cure for sickle-cell disease. Nature. 25 August 2021. Retrieved from:

[14] Frangoul H et al. CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia. N Engl J Med 2021; 384:252-260. January 21, 2021. Retrieved from:

[15] Newby, G.A., Yen, J.S., Woodard, K.J. et al. Base editing of haematopoietic stem cells rescues sickle cell disease in mice. Nature 595, 295–302 (2021). Retrieved from:'

[16] Thompson AA et al. Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia: Update from the Northstar Hgb-204 Phase 1/2 Clinical Study. Blood, volume 128, Issue 22. December 2, 2016. Retrieved from:

[17] Ledford H. CRISPR treatment inserted directly into the body for first time. Nature. 05 March 2020. Retrieved from:

[18] Stein R. A Gene-Editing Experiment Let These Patients With Vision Loss See Color Again. NPR-WNYC. September 29, 2021. Retrieved from:

[19] How CRISPR Is Changing Cancer Research and Treatment. National Cancer Institute. July 27, 2020. Retrieved from:

[20] Scudellari M. Self-destructing mosquitoes and sterilized rodents: the promise of gene drives. Nature. 09 July 2019. Retrieved from:

[21] Kyrou, K. et al. A CRISPR–Cas9 gene drive targeting doublesex causes complete population suppression in caged Anopheles gambiae mosquitoes. Nat Biotechnol 36, 1062–1066 (2018). Retrieved from:

[22] Javed MR et al. Current situation of biofuel production and its enhancement by CRISPR/Cas9-mediated genome engineering of microbial cells. Microbiological Research, Volume 219, Pages 1-1. 2019. Retrieved from:

[23] Gallegos J, 10 ways CRISPR will revolutionize environmental science. Alliance for Science. July 17, 2018. Retrieved from:

[24] World Malaria Report 2020-20 years of global progress & challenges. World Health Organization. 30 November 2020. Retrieved from:


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