What a decade of gene therapy research for sickle cell disease taught UCLA scientists
When Dr. Donald Kohn began performing bone marrow transplants in 1987, he saw firsthand the promise and limitations of available […]
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“Night and day”: Adventuring through childhood with newfound immune systems
Seven years ago, Jon and Alicia Langenhop learned that all three of their children suffered from the same devastating, one-in-a-million […]
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FDA approves gene therapy for severe leukocyte adhesion deficiency-I, a rare immune disorder
Dr. Donald Kohn has been developing gene therapies for rare pediatric immune disorders for over 30 years. This week, his […]
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Exposure to any germs could’ve killed her. 11 years later, she’s living a normal life thanks to a gene therapy treatment.
Two months after she was born, Eliana Nachem got a cough that wouldn’t go away. Three weeks later, she also […]
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Scientists are inventing treatments for devastating diseases. There’s just one problem.
This past spring, a biotech company announced the first use of a new gene-editing technology in people to fix an […]
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Gene therapy delivers lasting immune protection in children with rare disorder
An experimental gene therapy developed by researchers at UCLA, University College London and Great Ormond Street Hospital has restored and […]
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UCLA scientists advance gene therapy for deadly blood disorder alpha thalassemia major
Thanks to groundbreaking in-utero blood transfusion technology, what was once a fatal diagnosis in the womb can now result in […]
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A young girl with SCID becomes an advocate for herself and other
Seersha Sulack’s childhood has been anything but ordinary. For the first five years of her life, hospital rooms replaced playgrounds. […]
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Breathing new life: Inhalable gene therapy for cystic fibrosis
Imagine a future where treating cystic fibrosis is as simple as taking a deep breath. That’s exactly what a team […]
