The IGI and our collaborators have received a $4 million grant from the California Institute for Regenerative Medicine (CIRM) to develop CRISPR-Cas9 genome engineering into a cure for sickle cell disease (SCD).
This generous funding will support the fruitful ongoing collaboration between IGI scientific director Jacob Corn and his lab, physicians and sickle cell experts Mark Walters and David Martin of UCSF Benioff Children’s Hospital Oakland Research Institute (CHORI), and stem cell and gene therapy specialist Don Kohn of UCLA.
“We are very grateful for CIRM support, which enables us to establish clinical protocols for gene surgery to cure sickle cell disease,” says Dr. Corn. “CRISPR cures for genetic diseases are rapidly approaching the clinic, and our research will lay the groundwork for a clinical trial in SCD.” IGI researchers recently published proof-of-concept sickle gene editing in Science Translational Medicine, where they showed that the sickle cell mutation could be corrected in blood stem cells to near-therapeutic levels using the powerful CRISPR-Cas9 gene editing system. SCD is caused by a single-letter error in both copies of the hemoglobin gene, which causes red blood cell deformities and leads to blood vessel blockages, intense pain, and premature death. The disease can be cured by bone marrow transplant, but this procedure is risky and requires a matching donor.
The IGI team’s clinical approach will involve removing stem cells from the bone marrow of sickle cell patients, editing the mutated DNA code with the CRISPR-Cas9 system, and putting the corrected cells back into the patient, where they can persist and spawn healthy red blood cells. The funding from CIRM will enable the scientists to improve the efficiency of editing, scale up the process, and perform more extensive studies in animals to ensure safety and accuracy before moving into human clinical trials.
The grant is part of CIRM’s Translational Award program, which aims to move “the most promising projects out of the laboratory and into clinical trials in people.” Alongside the IGI work, the agency also funded future therapies against Alzheimer’s disease, cartilage damage, and osteonecrosis.
For a more in-depth perspective, read Dr. Corn’s thoughtful blog post on deploying gene editing to tackle sickle cell disease.
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