Transplantation Of Genome Edited Cells For Lysosomal Storage Disorders, 2024

The Mayo Clinic’s Center for Individualized Medicine and the Medical College of Wisconsin recently hosted a lecture in their Rare Disease Speaker Series, featuring Dr. Natalia Gomez-Ospina from Stanford University. Dr. Gomez-Ospina, an expert in medical genetics and lysosomal storage disorders, discussed innovative approaches to hematopoietic stem cell transplantation. She emphasized the potential of modifying patient cells and enhancing their recruitment to the brain to treat diseases that primarily affect the nervous system. This research aims to overcome significant challenges, such as the need for donor-derived cells and the difficulty of transporting these cells across the blood-brain barrier.

Dr. Gomez-Ospina’s work focuses on making bone marrow transplants safer and more effective for treating non-hematological diseases, particularly inherited metabolic disorders. By improving pre-transplant conditioning and leveraging genome editing, her team hopes to enhance the viability of these treatments for various conditions, including Hurler syndrome and Gaucher disease. Although much of the research is still in early stages, primarily involving cells and animal models, the advancements hold promise for future clinical applications, potentially benefiting many patients with currently untreatable genetic disorders.


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