New era of gene therapy treats baby with ultrarare disease
Dr Kiran Musunuru and Dr Rebecca Ahrens-Nicklas with patient KJ. Credit: Children’s Hospital of Philadelphia In a major step forward...
ultrarare
Gene therapy and bone marrow transplant alleviate ultra-rare genetic disease symptoms
SUMF1 positive cells in tissues following IT and combined IT/IV gene therapy. Credit: Communications Medicine (2025). DOI: 10.1038/s43856-025-00734-9 In two...
