A Breakthrough Using CRISPR’s Gene Editing Technology to Cure a Genetic Disorder

Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR), according to NCBI, are sequences that replicate and infect bacterial cells. CRISPR has been in the news recently, but this time it has definitely made headlines. “First-time” news according to an announcement today in Freethink.

CRISPR’s successful breakthrough marks a major “first” to use gene editing in the treatment of diseases. The excitement surrounding the treatment is due to CRISPR therapy curing a genetic disorder within the body and especially overcoming the many challenges during the last few years.

The Human Genome Research Center describes a genetic disorder as a change in the DNA sequence that differs from a normal sequence. Six thousand diseases can be attributed to genetic mutations.

What Did They Do and How Did They Do It?

This is the first time researchers have successfully treated a genetic disease by injecting CRISPR into the bloodstreams of patients in order to make corrections to the affected organs. The problem, until now, has been that although occurrences have been rare, CRISPR has occasionally been “off-target” and made potentially dangerous genetic changes.

Now, researchers at Regeneron Pharmaceuticals and Intellia Therapeutics have proven, by successfully targeting livers of patients with transthyretin amyloidosis, that CRISPR can travel through the bloodstream and accurately make edits in the targeted tissues.

How Did They Do It?

A CRISPR therapy called NTLA-2001 was injected into the bloodstreams of six patients who have transthyretin amyloidosis. The disease is a potentially fatal genetic disorder that produces a destructive protein. In this instance, CRISPR was designed to target and halt the production of the gene that makes that protein.

It only took one CRISPR injection and three patients who received the highest dose had their protein levels fall by up to ninety-six percent. This compares to the current treatment for transthyretin amyloidosis that only produces a drop of eighty-six percent but must be injected at regular intervals. The CRISPR therapy produced mild adverse events, and the team is monitoring the patients to find out whether the effect will be permanent.

The award-winning co-developer of CRISPR, Jennifer Doudna commented that accurate targeting, one of the major challenges, has been overcome.

Looking Forward

Researchers and providers are looking forward to using CRISPR injections in the treatment of disorders such as brain or heart diseases that are not an easy target.

Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia (AML) six years ago. During this period of partial remission, Rose researched investigational drugs to be prepared in the event of a relapse. Her husband died February 12, 2021 with a rare and unexplained occurrence of liver cancer possibly unrelated to AML.

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