When Dr. Donald Kohn began performing bone marrow transplants in 1987, he saw firsthand the promise and limitations of available treatments for life-threatening blood disorders. That experience planted the seed of a career-defining question: What if you could correct a […]... Read More
Seven years ago, Jon and Alicia Langenhop learned that all three of their children suffered from the same devastating, one-in-a-million genetic disorder. The constant, life-threatening infections and rashes that began weeks after each child’s birth, emergency room visits and hospital […]... Read More
Dr. Donald Kohn has been developing gene therapies for rare pediatric immune disorders for over 30 years. This week, his role in a clinical trial has culminated in the first-ever U.S. Food and Drug Administration-approved therapy for severe leukocyte adhesion […]... Read More
Two months after she was born, Eliana Nachem got a cough that wouldn’t go away. Three weeks later, she also started having runny stool, prompting a visit to her pediatrician.Eliana didn’t have allergies or a gastrointestinal condition; instead, tests pointed […]... Read More
This past spring, a biotech company announced the first use of a new gene-editing technology in people to fix an errant gene that causes a severe immune disorder. In June, a baby born with a life-threatening metabolic disorder was allowed […]... Read More
An experimental gene therapy developed by researchers at UCLA, University College London and Great Ormond Street Hospital has restored and maintained immune system function in 59 of 62 children born with ADA-SCID, a rare and deadly genetic immune disorder. Severe […]... Read More
Thanks to groundbreaking in-utero blood transfusion technology, what was once a fatal diagnosis in the womb can now result in live births. However, this medical advancement created a new challenge: a growing population of children born with that diagnosis — […]... Read More
Imagine a future where treating cystic fibrosis is as simple as taking a deep breath. That’s exactly what a team of pediatric physician-scientists at the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA is […]... Read More
Scientists at the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA have received $21.8 million in grants from the California Institute for Regenerative Medicine, the state’s stem cell agency, to develop and advance new […]... Read More
Days after their son was born in 2019, Andrea Fernandez and Kamil Guziak learned their new baby had a rare genetic disease and would be lucky to reach his second birthday. “You’re thinking, ‘How many pictures can I have with […]... Read More
Researchers from the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA will present their latest research and clinical trial results at the 66th American Society of Hematology (ASH) Annual Meeting and Exposition. The meeting, […]... Read More
Dr. Donald Kohn has been awarded a $14.7 million grant from the California Institute for Regenerative Medicine to develop a commercial manufacturing protocol for a groundbreaking gene therapy targeting ADA-SCID, a rare and life-threatening immune disorder. https://www.uclahealth.org/news/release/donald-kohn-awarded-147m-cirm-grant-advance-ada-scid-gene... Read More
Researchers at the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA have received more than $16 million in grants from the California Institute for Regenerative Medicine, the state’s stem cell agency, to advance new […]... Read More
CNN — Later this spring, a little girl in California who essentially has no immune system will receive a lifesaving treatment for “bubble boy disease” thanks to the persistence of a dogged group of parents, a pediatrician, a veteran newsman […]... Read More
For Evie Junior, living with sickle cell disease has been like running a marathon. “But it’s a marathon where as you keep going, the trail gets rockier and then you lose your shoes,” the 27-year-old said. “It gets harder […]... Read More
Celebrating the launch of The Centennial Campaign for UCLA, this video offers a glimpse into the miraculous work of Broad Stem Cell Research Center faculty member Dr. Donald B. Kohn and his impactful research to cure blood diseases.... Read More
Dr. Donald Kohn discusses a new breakthrough in stem cell research for sickle cell disease, and translating basic science discoveries to treatment.... Read More