Gene and Cellular Therapies for Leukodystrophies
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...
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MDPI AG,
2023-10-01T00:00:00Z.
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