Cookies

Like most websites The Medicine Maker uses cookies. In order to deliver a personalized, responsive service and to improve the site, we remember and store information about how you use it. Learn more.
Manufacture Advanced Medicine

The CRISPR Toolbox

Since CRISPR (clustered regularly interspaced short palindromic repeats) gene editing was discovered in 2012, its ability to make precise and permanent changes in the DNA of both animals and plants has been generating excitement. The focus of medical science research to-date has been on diseases caused by a single gene mutation, such as sickle cell anemia (SCA) and beta thalassemia, and the improvement of anti-tumor immunotherapy. The first use of an investigational ex vivo CRISPR-based therapy to treat SCA and beta thalassemia is already underway in at least two patients in clinical trials. 

CRISPR gene editing makes use of enzymes, particularly nucleases, that have been programmed to target specific sequences in the genome and then introduce edits. However, DNA cleavage and editing may occur at additional off-target sites in the genome that have similar but different DNA sequence from that of the intended site.

Read the full article now

Log in or register to read this article in full and gain access to The Medicine Maker’s entire content archive. It’s FREE and always will be!

Login

Or register now - it’s free and always will be!

You will benefit from:

  • Unlimited access to ALL articles
  • News, interviews & opinions from leading industry experts
  • Receive print (and PDF) copies of The Medicine Maker magazine
Register

Or Login via Social Media

By clicking on any of the above social media links, you are agreeing to our Privacy Notice.

About the Author

Garrett Rettig

Research Scientist at Integrated DNA Technologies (IDT)

Register to The Medicine Maker

Register to access our FREE online portfolio, request the magazine in print and manage your preferences.

You will benefit from:

  • Unlimited access to ALL articles
  • News, interviews & opinions from leading industry experts
  • Receive print (and PDF) copies of The Medicine Maker magazine

Register