Lysosomal Storage Diseases: Stem Cell-Based Cell- and Gene-Therapy|
Transplantation of hematopoietic stem cells, bone marrow stem cells and umbilical cord blood-derived stem cells offer effective but limited efficacy for patients suffering from Krabbe disease, mucopolysaccharidosis VII and adrenal leukodystrophy but in other lysosomal storage diseases (LSDs) they are ineffective. Cell-based gene therapies for LSDs bridge the application of enzyme replacement therapy and gene therapy and are important direction to pursue in the future. [Cell Transplant]
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