Gene therapy for lysosomal storage disorders: recent advances for metachromatic leukodystrophy and mucopolysaccaridosis I.

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Term Occurence Count Dictionary
Fabry disease 2 endocrinologydiseases
glycogen storage disease 1 endocrinologydiseases
leukodystrophy 6 endocrinologydiseases
metachromatic leukodystrophy 4 endocrinologydiseases
mucopolysaccharidosis 2 endocrinologydiseases

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Fabry disease 6072 different LSDs, like Pompe disease (Chakrapani et al [16]), type I Gaucher disease (Gonzalez et al [33]), Fabry disease (Alfadhel and Sirrs [4]) and MPS, type I (Laraway et al [44]), II (da Silva et al [23]), IVA (Tomatsu
Fabry disease 33975 LSDs liver damage, wild-type mice were treated with AAV8-ZFN encoding either human α-galactosidase A ( Fabry disease ), acid β-glucosidase (Gaucher diseases), iduronate-2 sulfatase (Hunter syndrome), or α-l-iduronidase
glycogen storage disease 3388 into mucopolysaccharidoses, mucolipidoses, glycoproteinoses, sphingolipidoses, oligosaccharidosis and glycogen storage disease s (Futerman and van Meer [30]). Recent understanding of the molecular basis of these diseases has allowed
leukodystrophy 125 Inherited Metabolic DiseaseGene therapy for lysosomal storage disorders: recent advances for metachromatic leukodystrophy and mucopolysaccaridosis IRachele PenatiFrancesca FumagalliValeria CalbiMaria Ester BernardoAlessandro
leukodystrophy 1755 HSC-based gene therapy approaches for the treatment of LSDs with particular focus on metachromatic leukodystrophy (MLD) and mucopolysaccharidosis type I (MPS-I).IntroductionLysosomal storage diseases (LSDs) are rare
leukodystrophy 4619 (MPS-I) (OMIM # 607014), caused by a deficiency of the enzyme alpha-L-iduronidase, and metachromatic leukodystrophy (MLD, OMIM # 250100), due to deficiency of arylsulfatase A (ARSA) enzyme, as models of LSD with nervous
leukodystrophy 19297 the nervous system.So far clinical trials using LV transduced HSCs are ongoing in patients with adreno leukodystrophy (ALD), metachromatic leukodystrophy (MLD), β-thalassemia, Wiskott-Aldrich syndrome (WAS), severe combined
leukodystrophy 19333 trials using LV transduced HSCs are ongoing in patients with adrenoleukodystrophy (ALD), metachromatic leukodystrophy (MLD), β-thalassemia, Wiskott-Aldrich syndrome (WAS), severe combined immunodeficiency (SCID) caused
leukodystrophy 24330 population.Promising results have been obtained in preclinical studies for the treatment of globoid cell leukodystrophy (GLD), which is caused by mutation in the gene encoding galactocerebrosidase (GALC). Gentner et al (Gentner
metachromatic leukodystrophy 111 Journal of Inherited Metabolic DiseaseGene therapy for lysosomal storage disorders: recent advances for metachromatic leukodystrophy and mucopolysaccaridosis IRachele PenatiFrancesca FumagalliValeria CalbiMaria Ester BernardoAlessandro
metachromatic leukodystrophy 1741 scientific advances in HSC-based gene therapy approaches for the treatment of LSDs with particular focus on metachromatic leukodystrophy (MLD) and mucopolysaccharidosis type I (MPS-I).IntroductionLysosomal storage diseases (LSDs) are rare
metachromatic leukodystrophy 4605 mucopolysaccharidosis type I (MPS-I) (OMIM # 607014), caused by a deficiency of the enzyme alpha-L-iduronidase, and metachromatic leukodystrophy (MLD, OMIM # 250100), due to deficiency of arylsulfatase A (ARSA) enzyme, as models of LSD with nervous
metachromatic leukodystrophy 19319 far clinical trials using LV transduced HSCs are ongoing in patients with adrenoleukodystrophy (ALD), metachromatic leukodystrophy (MLD), β-thalassemia, Wiskott-Aldrich syndrome (WAS), severe combined immunodeficiency (SCID) caused
mucopolysaccharidosis 1780 approaches for the treatment of LSDs with particular focus on metachromatic leukodystrophy (MLD) and mucopolysaccharidosis type I (MPS-I).IntroductionLysosomal storage diseases (LSDs) are rare inherited metabolic disorders
mucopolysaccharidosis 4489 [81], Jeyakumar et al [38]).This review will focus in particular on two autosomal recessive disorders, mucopolysaccharidosis type I (MPS-I) (OMIM # 607014), caused by a deficiency of the enzyme alpha-L-iduronidase, and metachromatic

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