Gene therapy for mucopolysaccharidoses: in vivo and ex vivo approaches

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Term Occurence Count Dictionary
metachromatic leukodystrophy 1 endocrinologydiseases
mucopolysaccharidosis 1 endocrinologydiseases
leukodystrophy 2 endocrinologydiseases
lysosomal storage disease 1 endocrinologydiseases

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leukodystrophy 22926 in mouse models of LSD by transplanting LV-transduced HSCs has also been reported for metachromatic leukodystrophy (MLD) and globoid cell leukodystrophy (GLD) [[55]–[57]]. Using this approach, microglia replacement
leukodystrophy 22964 transplanting LV-transduced HSCs has also been reported for metachromatic leukodystrophy (MLD) and globoid cell leukodystrophy (GLD) [[55]–[57]]. Using this approach, microglia replacement by gene-corrected cells provided a population
lysosomal storage disease 1744 vision for the future in the medical community.BackgroundMucopolysaccharidoses (MPS) are a group of 11 lysosomal storage disease s (LSDs) caused by a deficiency of enzymes involved in lysosomal breakdown of the glycosaminoglycans
metachromatic leukodystrophy 22912 manifestations in mouse models of LSD by transplanting LV-transduced HSCs has also been reported for metachromatic leukodystrophy (MLD) and globoid cell leukodystrophy (GLD) [[55]–[57]]. Using this approach, microglia replacement
mucopolysaccharidosis 12456 (SGSH) has been planned by Lysogene.Table 1Clinical trials of ex-vivo and in-vivo gene therapy (GT) in mucopolysaccharidosis (MPS)DiseaseStudy phaseType of vectorRouteNo. of treated patientsOutcomeSponsorIn vivo MPS IIIAI/IIAAVrh.10-SGSH-IRES-SUMF1IC4Improvement

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