Cell and gene therapies are exciting areas of research because of the potential to heal diseased or damaged tissues rather
than to replace them. Cell therapies are already being delivered for a variety of conditions, and scientists hope to
eventually apply them to treat liver disease, diabetes, neural disorders, renal failure and other chronic conditions.
Some of the projects at Wake Forest Institute for Regenerative Medicine (WFIRM) include:
Although there are medications to compensate for the loss of female sex hormones, they often aren’t recommended for long-term use because of the increased risk of heart disease and breast cancer. Institute researchers are working toward a cell-based or tissue-based hormone therapy—essentially an artificial ovary to deliver hormones in a more natural manner than drugs. Their strategy involves engineering ovarian tissue that integrates with the body using the technique of encapsulating ovarian cells inside a thin membrane that allows oxygen and nutrients to enter, but would prevent the patient from rejecting the cells. With this approach, functional ovarian tissue from donors would be used to engineer bioartificial ovaries for women with impaired ovarian function.
There is currently no cure for hemophilia A, the most common inherited coagulation disorder that causes prolonged bleeding after an injury and can result in joint and organ damage and life-threatening internal bleeding. Institute researchers are exploring a combined cell and gene therapy for the disorder. Their strategy is to engineer mesenchymal stem cells, which have the ability to migrate to sites of injury and inflammation, to produce high levels of factor VIII, the clotting protein missing in people with hemophilia. The cells—acting as a carrier for the gene—would then be transplanted into the patient. In an animal model of hemophilia A, the treatment stopped ongoing bleeding and existing joint damage was reversed.
A potential new therapy for urinary incontinence in women, developed by institute scientists, is being evaluated in a clinical study. Millions of women are affected by the accidental leakage of urine, such as when they sneeze or cough. The treatment involves taking a small sample of muscle tissue from patients, growing the cells in the lab and then injecting the patient’s own muscle cells into the urinary sphincter, the circular muscle that controls urine flow. The treatment has been successful in animal studies and the clinical study will test its effectiveness in women.