Sickle cell treatment? CRISPR gene editing shows promise

sickle
Sickle cell patient. Source: Marcus Yam, Los Angeles Times

A gene editing technology called CRISPR/Cas9 repaired 20 to 40 percent of stem and progenitor cells obtained from sickle cell disease patients, scientists report, suggesting such an approach might offer a way of treating this disease.

Sickle cell anemia is the most severe type of sickle cell disease, which is a group of inherited conditions that affect hemoglobin, the molecule in red blood cells that transports oxygen throughout the body. A single mutation in the gene that helps to make hemoglobin alters red blood cells to a sickle, crescent shape. These cells die prematurely, leading to anemia, and may also get stuck in small blood vessels, blocking blood flow.


Researchers injected these altered cells into the bone marrow of mice with defective immune systems and determined how many retained the edit after 19 weeks. “The rate of repair remained stable, which is great,” [said Gang Bao, PhD, a member of the research team and a bioengineer at Rice.]

Regarding their clinical significance and whether repairing as much as 40 percent of the cells is enough to cure the disease, Bao noted that more work is needed. “We’d like to say, ‘Yes,’” he said, “but we don’t really know yet. That’s something we hope to learn from an eventual clinical trial.”

ADVERTISEMENT

Read full, original post: CRISPR/Cas9 Gene Editing Shows Potential to Treat Sickle Cell Disease, Study Reports

Outbreak Daily Digest
Biotech Facts & Fallacies
GLP Podcasts
Infographic: Here’s where GM crops are grown around the world today

Infographic: Here’s where GM crops are grown around the world today

Do you know where biotech crops are grown in the world? This updated ISAAA infographics show where biotech crops were ...
News on human & agricultural genetics and biotechnology delivered to your inbox.
glp menu logo outlined

Newsletter Subscription

* indicates required
Email Lists
Send this to a friend