![]() ![]() Last fall, Kohn, director of the UCLA Human Gene Medicine Program, reported the results of what he calls Version 2.0 of the therapy. The modified cells were then infused back into the babies to help restore their immune systems. Their cells were cultured with viral vectors, which transferred a normal copy of the ADA gene into the stem cells. He and his colleagues isolated stem cells from the umbilical-cord blood of three newborns diagnosed with the disease before birth. Donald Kohn.Two decades ago, Kohn, then at Children’s Hospital Los Angeles, performed the world’s first gene therapy on newborns with ADA-deficient SCID. “My hope is that this can be a meaningful first step toward regenerative medicine for the eye.” Bursting the Bubbleĭr. “Our results have been so positive in terms of safety, that the Food and Drug Administration granted us permission to open up another cohort in the study: those with better vision,” Schwartz said. As a result of that early success, Schwartz and his colleagues have transplanted RPE cells in 18 more patients and have expanded the trial to include four other top eye institutes. Neither woman suffered side effects such as retinal detachment, eye inflammation or abnormal cell growth. The woman with macular degeneration, for example, went from being unable to read any letters on a visual-acuity chart to discerning five letters. Four months after the injections, both patients felt they saw more clearly. “Both of these diseases have, as a final common pathway, the death of the RPE.” The RPE had been considered “low-hanging fruit” for an embryonic stem cell trial, he noted, in part, because the cells are terminally differentiated, can be accessed surgically and have no synaptic connections. When those cells go, vision goes,” said Schwartz, Ahmanson Professor of Ophthalmology and chief of the Retina Division at UCLA’s Jules Stein Eye Institute. ![]() Portraits by Ann Johansson.RPE cells, which form a supportive layer beneath the retina, “are 100 percent critical for vision. ![]() 1 cause of blindness in the developed world, and a woman with Stargardt’s macular dystrophy, a progressive vision disorder that can lead to blindness by the third or fourth decade of life - received relatively low doses of hESC-derived retinal pigment epithelial (RPE) cells transplanted into the space beneath the retina of one eye each.ĭr. Both patients - a woman with dry age-related macular degeneration, the No. Steven Schwartz and his colleagues reported the first safe clinical use of hESC-derived cells in two legally blind patients. Their promise is finally beginning to be realized in January 2012, UCLA retinal specialist Dr. For that reason, human embryonic stem cells (hESCs) are considered ideal tools for regenerative medicine. Whereas adult stem cells, which are found in particular organs of the mature body - the bone marrow, for example, or the brain - can only produce the specialized cells for that particular tissue type, embryonic stem cells derive from a far earlier point in development and thus have the potential to differentiate into every type of cell in the body. ![]() Stem cells come in two forms, adult and embryonic. The state agency was established in 2004 to fund translational stem-cell research at institutions throughout California with the goal of developing new therapies for deadly diseases and disorders. Much of the work being done is supported by both private and institutional sources, including grants approaching $190 million from the California Institute for Regenerative Medicine (CIRM). At the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, more than 200 faculty members are working to translate the promise of stem cells into viable treatments for some of society’s most vexing medical conditions, including cancer, heart disease, immune disorders, Alzheimer’s and Parkinson’s diseases, autism, blindness and diabetes. Combine those two superpowers, and you’ve got the proverbial medical magic bullet. They have two unique abilities: They can proliferate virtually without limit to produce an essentially infinite supply of their unspecialized cellular selves, and they can differentiate to produce any other cell types that can be used to repair or replace worn-out or damaged tissues. To see more, click here. Stem cells are the body’s “master” cells. Shown above are some of the stem cells derived by scientists at the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA. ![]()
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