CRISPR-Cas9 in Patients with Sickle Cell

The largest-ever research grant for a study using the gene-editing technology CRISPR-Cas9 in patients with sickle cell anemia has been approved. UCSF Benioff Children’s Hospital Oakland will be the first institution to use CRISPR-Cas9 technology in humans.

The four-year study is under the direction of Dr. Mark Walters, a UCSF professor of pediatrics who is leading the clinical trial and gene-editing project.

The study, conducted in collaboration with researchers at UCLA and UC Berkeley, aims to alter the programming of a part of the blood system to produce healthy red blood cells instead of sickle-shaped red blood cells. The initial phases of the study will focus on safety to see how the cells behave once they are back in the body’s circulation.

“What we’re going to do is remove a small portion of the blood system, change its programming to no longer make sickle red blood cells, but healthy red bloods cells, and then return them to the circulation where they’ll begin to grow and build new blood cells,” Dr. Walters said on KCBS Radio’s “As Prescribed.”

UCSF researchers would enroll nine patients in the first phase, primarily adults who are at risk of dying from sickle cell disease if immediate treatment is not available.

If the trial is successful, the team believes patients could become donors themselves instead of waiting for a matching donor before blood transfusions and bone marrow transplants. In addition, the treatment would become more affordable and accessible to those who need it.

Source: MSN