| Article ID | Journal | Published Year | Pages | File Type |
|---|---|---|---|---|
| 10833743 | Molecular Genetics and Metabolism | 2012 | 8 Pages |
Abstract
⺠We provide an overview of developing treatment options in patients with MLD. ⺠Data concerning hematopoietic stem cell transplantation are discussed. ⺠Enzyme replacement and gene therapy represent novel therapeutic strategies in MLD. ⺠Data concerning add-on treatment options still on experimental level are also mentioned.
Keywords
LSDFGEGvHDFormylglycine-generating enzymeArylsulfatase AUCBTlentiviralMLDAAVMSCTM6PBMTHSCTARSAenzyme replacement therapyLysosomal storage diseaseGraft-versus-host diseaseCNSBBBcentral nervous systemperipheral nervous systemMetachromatic leukodystrophymannose-6-phosphateERTadeno-associated viralUmbilical cord blood transplantationmesenchymal stem cell transplantationBone marrow transplantationHematopoietic stem cell transplantationGene therapyPNS
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Authors
Spyros P. Batzios, Dimitrios I. Zafeiriou,