Improved Brain Pathology and Progressive Peripheral Neuropathy in a 15 Year Old Survivor of Infantile Krabbe Disease Treated With Umbilical Cord Transplantation

OBJECTIVE: Krabbe disease is a fatal leukodystrophy caused by deficiency in galactocerebrosidase enzyme activity. The only currently available therapy is hematopoietic stem cell transplantation with bone marrow or umbilical cord blood (UCBT), which leads to increased lifespan and functional abilitie...

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Detalles Bibliográficos
Autores principales: Kofler, Julia, Beltran-Quintero, Maria L., Rugari, Anne, Zuccoli, Giulio, Klotz, Sarah, Escolar, Maria L.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2022
Materias:
Molecular Neuroscience
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9368320/
https://www.ncbi.nlm.nih.gov/pubmed/35966016
http://dx.doi.org/10.3389/fnmol.2022.888231

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