Rice team gains ground in CRISPR cure for sickle cell, but challenges remain

CRISPR
CRISPR is an attracting treatment for sickle cell disease, which is caused by a single mutation. (Image: Stephen Dixon)

Researchers led by Rice University used CRISPR to correct the sickle cell mutation in up to 40% of stem cells taken from affected patients. While promising, the work also highlights the hurdles that must be cleared before gene editing can become a reality for patients with blood disorders.

CRISPR is an appealing approach for sickle cell disease, which is caused by a single mutation in the gene that codes for beta-globin, a component of red blood cells. Gang Bao, a bioengineering professor at Rice University, detailed a series of tests exploring the use of CRISPR to fix this mutation at the American Association for the Advancement of Science’s annual meeting.

The researchers zeroed in on stem cells and progenitor cells, which can differentiate into a more limited range of cell types than stem cells: "The idea is to correct that particular mutation, and then stem cells that have the correction would differentiate into normal blood cells, including red blood cells. Those will then be healthy blood cells,” Bao said.

Infographic Download

Reducing Time to Clinic for Your Biomedical Applications

Gelatin methacryloyl (GelMA)-based biomaterials have been widely used in various biomedical research applications due to their suitable biological properties and tunable physical characteristics. Especially over the past 5 years, GelMA-oriented research and patent applications have been growing exponentially, and many of these research concepts are now being translated towards the clinic. Suitable GelMA biomaterials are therefore indispensable to keep pace with the newest medical innovations.

Download to learn more about the benefits of GelMA in various biomedical applications and how X-Pure® GelMA can help you in your developments.

RELATED: CRISPR a potential cure for sickle cell disease

Alongside colleagues from Baylor College of Medicine and Texas Children’s Hospital, Bao’s team gathered stem and progenitor cells from patients with sickle cell disease and used CRISPR to correct the mutation. They were able to repair between 20% and 40% of the cells.

A team at Matt Porteus’s lab at Stanford then injected the edited cells into the bone marrow of mice, where they persisted after 19 weeks.

"The rate of repair remained stable, which is great," Bao said.

RELATED: Bluebird’s tweaked sickle cell gene therapy clears early test

While a step in the right direction, the research highlights some obstacles. These include tweaking the method to prevent unintended off-target effects and trying to repair a higher proportion of stem cells, as the team is unsure if correcting 40% is enough to cure a patient.

Gene therapy for sickle cell and other blood disorders has been ramping up. Vertex Pharmaceuticals teamed up with CRISPR Therapeutics in 2015, and recently licensed the first CRISPR-based treatment to come out of the partnership: a therapy for beta thalassemia and sickle cell.

Suggested Articles

Spinal Elements, maker of a wide range of implants and products for minimally invasive spine procedures, has filed a $100 million IPO.

United Airlines will begin providing COVID-19 screening tests for passengers, allowing those who test negative to skip local quarantine requirements.

Galecto picked up $64 million to push its lead lung disease treatment toward an approval in Europe and fund midstage studies for its other programs.