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What is regenerative medicine?

Regenerative medicine is a rapidly growing field that develops methods to regrow, repair or replace damaged or diseased cells, organs or tissues. It often includes the generation and use of therapeutic stem cells, tissue engineering and the production of artificial organs. It often includes the generation and use of therapeutic stem cells or gene therapy, tissue engineering and the production of artificial organs.

What are stem cells?

Stem cells are immature cells that have the ability to turn into other cell types. They are found in large numbers in fetal tissues, but also in adult tissues. Induced-pluripotent stem cells are created in a lab from mature cells—often blood or skin cells—by reverting them to an immature state. 

What is a stem cell line?

A stem cell line is a population of cells that can reproduce repeatedly without maturing. Stem cell lines come from a variety of sources and are maintained in a lab. They can be stored frozen and then re-cultured for later use. 

Why are stem cells useful in medicine?

Stem cells can be used to grow replacement cells. Transplants of blood and bone marrow stem cells have been used for more than 40 years to treat leukemia, lymphoma, and other blood disorders. More recently, scientists are attempting to use stem cells to grow new tissues, and even organs consisting of multiple cell types. Scientists are also exploring the possibility of triggering dormant stem cells in the body to grow and replace tissues damaged by disease or injury.   

Why are stem cells useful in research?

Stem cells are helping scientists better understand cell function and tissue development, under normal conditions and during disease. Stem cells theoretically can provide unlimited numbers of mature human cells, which can be used for testing potential drugs or other therapies. 

Are stem cells used to treat eye disease and vision disorders?

Stem cell-based treatments for the eye are undergoing testing in clinical trials.  

Researchers at the NEI are testing the safety of treating people with age-related macular degeneration (AMD) using tissues made from patient-derived stem cells. The tissue they are making is called retinal pigment epithelium (RPE). The dry form of AMD causes the breakdown of RPE, which leads to the death of the light-sensing photoreceptors and, in turn, the loss of central vision that is crucial for things like reading and driving. Learn more about the NEI clinical trial

image explaining how iPSC works
Researchers will take a patient’s own blood cells, and in a lab, convert them into iPS cells capable of becoming any type of cell in the body. The iPS cells are then programmed to become retinal pigment epithelial cells, the type of cell that dies early in the geographic atrophy form of AMD. Credit: NEI

NEI-funded researchers at Harvard Medical School are conducting a clinical trial to test restoring the eye’s clear outer layer called the cornea using patient-derived stem cells Participants in the trial suffered chemical burns in one eye that caused the cornea to become cloudy. Using a procedure called cultivated autologous limbal epithelial cell transplantation (CALEC), the researchers remove a limited number of stem cells from the patient’s healthy eye, grow them into tissue in the lab, and then graft the resulting tissue into the injured eye.  Click here to view a video on this cutting-edge therapy for cornea damage. 

Concerned about stem cell clinics?

The International Society for Stem Cell Research (ISSCR), a non-profit professional stem cell research organization, is concerned that stem cell therapies are being sold around the world before they have been proven safe and effective. To help combat this issue, ISSCR has developed a patient handbook to help you make the best decisions for your treatments.

Click here for information about stem cell-based treatments for AMD from the International Society for Stem Cell Research.  

Want to know more about clinical trials at NIH?

Learn more about clinical trials by visiting NIH Clinical Trials and You website

To find out about clinical studies supported by at the NEI Eye Clinic, visit the NIH Clinical Center website or call 1-800-411-1222.  Talk with your doctor about whether participating in a clinical trial is right for you. 

Additional Patient Resources About Gene Therapy

A compilation of third-party resources that are highly relevant to patient groups that want to learn more about gene therapy.

National Organization for Rare Disorders (NORD)

Gene Therapy: Your Questions Answered

NORD’s RareEDU™ released this video, Gene Therapy: Your Questions Answered, in order to address a vital topic to today's rare disease community. The goal of this video is to address the questions, hopes and concerns that patients and caregivers, across many different diseases, have about gene therapy. Since more than 80% of rare diseases are believed to be genetic, this video will serve as a helpful resource for the rare disease community.

Global Genes

Do you or a loved one have a rare genetic disease? A Guide to Gene Therapy from Global Genes can serve as a great toolkit. 

U.S. Food and Drug Administration (FDA)

Gene therapy is the process of replacing defective genes with healthy ones, adding new genes to help the body fight or treat disease, or deactivating problem genes. It holds the promise to transform medicine and create options for patients who are living with difficult, and even incurable, diseases. Learn how this innovative therapy works with by viewing Gene Therapy Inside Out

FDA update from Peter Marks .D., Ph.D., Director, Center for Biologics Evaluation and Research on Advancing the Development of Safe and Effective Regenerative Medicine Products

Genetic Science Learning Center

American Society for Gene and Cell Therapy (ASGCT) and NORD

ASGCT and the National Organization for Rare Disorders (NORD) have partnered to produce a series of five patient education webinars.  View the Gene Therapy Patient Education Webinars.

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Last updated: June 26, 2023