Gene and Cellular Therapies for Leukodystrophies

• Main Authors: Fatima Aerts-Kaya (Author), Niek P. van Til (Author)
• פורמט: ספר
• יצא לאור: MDPI AG, 2023-10-01T00:00:00Z.
• נושאים: article
• גישה מקוונת: Connect to this object online.

Leukodystrophies are a heterogenous group of inherited, degenerative encephalopathies, that if left untreated, are often lethal at an early age. Although some of the leukodystrophies can be treated with allogeneic hematopoietic stem cell transplantation, not all patients have suitable donors, and ne...