does crispr gene editing have promise as a cure for sickle cell disease 94562 1 - Does CRISPR Gene Editing Have Promise as a Cure for Sickle Cell Disease?
May 27, 2022

Sickle cell disease is a painful condition afflicting people around the world. It’s an inheritable condition, with a genetic mutation that makes individuals’ red blood cells occur in the curved shape of a sickle.

The sickle shape tends to make blood cells clump together in the patients’ thinner blood vessels. This causes severe pain and leads to organ damage. While there are treatments to modify the impact of the disease, there is presently no cure.

Now, researchers in search of a cure for this debilitating disorder are turning their attention to CRISPR gene editing to see if they can make a positive difference in their patients’ lives.

Focus on Sickle Cell Disease

Sickle cell disease afflicts about 100,000 people in the United States today, according to Healio. With sickle cell disease, a patient’s hemoglobin can’t transport oxygen through the blood as efficiently as it would in a healthy person’s body.

Patients see a lifespan decrease of 30 years compared to those not afflicted by this genetic disorder. The painful condition affects Black people the greatest.

Work With CRISPR Gene Editing Begins at UCSF in Oakland, California

Sickle cell disorder, when inherited from one’s relatives, is the result of a single mutation in the genetic code. The role of CRISPR is to actually edit the genes in a person’s cells while they are still living and then put them back in the body to effect a potential cure.

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At UCSF Benioff Children’s Hospital Oakland, researchers are using an $8.6 million grant from the NIH Cure Sickle Cell Initiative and another $8.4 million grant from the California Institute of Regenerative Medicine to create a four-year clinical trial using CRISPR gene editing to potentially find a cure for sickle cell disorder.

An important aspect of the grants is that they help cover work done on patients, which insurance companies do not pay for. Work is done through a University of California Research consortium that also involves UCLA and UC Berkley.

In Oakland, the clinical trial will be the first occasion when technicians use CRISPR to edit genes in an effort to fix a beta-globin genetic mutation, fixing them in the patient’s cells.

The project began after principal investigator Mark Walters, MD met with CRISPR pioneers Jennifer Doudna and Emmanuelle Charpentier, who first described the novel gene editing technique in 2012, and for which they won the Nobel Prize in chemistry in 2020.

CRISPR in the Clinic

The UC researchers will commence the trial by removing blood stem cells from a patient. Then, in UCLA’s gene manufacturing lab, technicians will apply electrical pulses to make small holes appear in the membrane of the cells. Then, the CRISPR/Cas9 platform moves into the patient’s cells, where it edits the genes to remove the mutation.

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At this point, it’s time to infuse these newly edited cells back into the patient’s body. The effort through the University of California system is meant to be a proof-of-concept, showing that blood stem cells from sickle cell patients can actually be edited successfully to repair the pain-causing and lifespan-reducing genetic mutation.

A Future Cure With Clustered Regularly Interspaced Short Palindromic Repeats in the Clinic to Battle Sickle Cell Disease?

Scientists will not jump to say that CRISPR gene editing constitutes a complete cure for patients with sickle cell disease, as more clinical trials and tests will be needed to ascertain the treatment’s effectiveness as well as safety. But the initial results from researchers are showing some signs of improvement, and that’s something that patients can hold onto as they await a definitive cure.

 

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