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Cell and Gene Therapies

Cell therapies are an exciting area of research because in some cases it may be easier to heal diseased/damaged tissues rather than to replace them. Cell therapies apply living cells to an organ or tissue to promote healing and regeneration from within. Cell therapies are being delivered today for cartilage reconstruction, bone reconstruction, and in inflammatory and immune response problems. In the future, cell therapies hold promise for treating liver disease, diabetes, neural disorders, renal failure and other chronic conditions.

Gene therapies are being explored for disorders that result when genes are altered so that the encoded proteins are unable to carry out their normal functions. Gene therapy is a technique for correcting defective genes responsible for disease development. Researchers may use one of several approaches for correcting faulty genes. In the U.S., a variety of gene therapy clinical studies are under way. 

Research Initiatives

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	Learn more about our project to develop a cell-based hormone therapy.
	Stem cells found in placenta show promise as a treatment for cystic fibrosis.
	Our scientists are trying several new strategies in projects to help restore the body's natural ability to produce insulin.
	There is currently no cure for the bleeding disorder hemophilia, but early success using gene therapy in preclinical trials points to the possibility of using the same approach in people.
	A project to develop a gene therapy treatment for myotubular myopathy may one day benefit children born with this often fatal disease.