A New World of Cell and Gene Therapies
A New World of Cell and Gene Therapies
Researchers at the Miller School of Medicine have been rigorously studying how stem cells (immature cells that can differentiate into many tissue types) can rejuvenate damaged tissues. More recently, gene editing has emerged as a way to potentially counteract genetic risk and treat various common diseases. These technologies are not yet ready for the clinic, but they are getting close.
Joshua Hare, M.D., the Louis Lemberg Professor of Medicine who directs the Donald Soffer Endowed Program in Regenerative Medicine, has spent many years trying to understand how stem cells could improve cardiovascular disease, pulmonary fibrosis, aging-related frailty and other conditions. His lab has been particularly focused on mesenchymal stem cells, which can become bone, fat, cartilage, muscle and other tissues, and also have powerful tissue-healing effects.
Dr. Hare is helping run two studies testing whether mesenchymal stem cells, derived from umbilical cords, can help patients with heart failure. One is studying ischemic cardiomyopathy – heart failure caused by coronary artery disease. The other is addressing dilated cardiomyopathy, or heart failure from other causes.
“The dilated cardiomyopathy trial will enroll 108 patients, and the ischemic cardiomyopathy study will enroll 60,” Dr. Hare said. “These are significant studies, and we hope they’ll be enough to push us closer to the finish line and get these treatments to patients.”
Harnessing the amazing power of stem cells
Tune in to hear Dr. Hare discuss the amazing power of stem cells.
Stem cell technologies offer another approach that can improve care: organoids. Researchers can now take a patient’s skin cells (or, really, any cells) and revert them to their immature state: induced pluripotent stem cells. In turn, these cells can be differentiated into virtually any tissue, allowing scientists to make small, 3D organoids.
“By studying an organoid, we can potentially determine exactly what’s wrong with a patient,” Dr. Hare said. “This could really help us guide care, especially when the problem is genetic.”
Gene therapies, particularly gene editing, hold vast promise to correct the genetic defects that may be driving heart disease in some patients. When a gene is not functioning properly, a gene therapy could insert that missing sequence. A gene editing technique called CRISPR/Cas9 could actually fix an existing gene to help it perform better.
“In the lab, we can turn a good gene on; we can also turn an undesirable gene off,” Dr. Hare said. “We’re hoping to use CRISPR to genetically treat a number of common cardiovascular conditions, such as high blood pressure and elevated cholesterol. We’re going to be the leading center in Florida for that.”
“By studying an organoid, we can potentially determine exactly what’s wrong with a patient, this could really help us guide care, especially when the problem is genetic.”
