History, genetic, and recent advances on Krabbe disease
ACE Graziano, V Cardile - Gene, 2015 - Elsevier
Krabbe disease or globoid cell leukodystrophy is one of the classic genetic lysosomal
storage diseases with autosomal recessive inheritance that affects both central and …
storage diseases with autosomal recessive inheritance that affects both central and …
Globoid cell leukodystrophy (Krabbe's disease): update
K Suzuki - Journal of child neurology, 2003 - journals.sagepub.com
The classic globoid cell leukodystrophy (Krabbe's disease) is caused by genetic defects in a
lysosomal enzyme, galactosylceramidase. It is one of the two classic genetic …
lysosomal enzyme, galactosylceramidase. It is one of the two classic genetic …
[HTML][HTML] Krabbe disease: New hope for an old disease
AM Bradbury, ER Bongarzone, MS Sands - Neuroscience letters, 2021 - Elsevier
Krabbe disease (globoid cell leukodystrophy) is a lysosomal storage disease (LSD)
characterized by progressive and profound demyelination. Infantile, juvenile and adult-onset …
characterized by progressive and profound demyelination. Infantile, juvenile and adult-onset …
Genetic ablation of acid ceramidase in Krabbe disease confirms the psychosine hypothesis and identifies a new therapeutic target
Infantile globoid cell leukodystrophy (GLD, Krabbe disease) is a fatal demyelinating disorder
caused by a deficiency in the lysosomal enzyme galactosylceramidase (GALC). GALC …
caused by a deficiency in the lysosomal enzyme galactosylceramidase (GALC). GALC …
Enhanced efficacy and increased long-term toxicity of CNS-directed, AAV-based combination therapy for Krabbe disease
Infantile globoid cell leukodystrophy (GLD, Krabbe disease) is a demyelinating disease
caused by the deficiency of the lysosomal enzyme galactosylceramidase (GALC) and the …
caused by the deficiency of the lysosomal enzyme galactosylceramidase (GALC) and the …
Allogeneic stem cell transplantation for the treatment of lysosomal and peroxisomal metabolic diseases
W Krivit - Springer seminars in immunopathology, 2004 - Springer
This is a review of the clinical responses and prospectus of new therapies following use of
allogeneic hematopoietic stem cell transplantation for the treatment of the following …
allogeneic hematopoietic stem cell transplantation for the treatment of the following …
Mechanism-based combination treatment dramatically increases therapeutic efficacy in murine globoid cell leukodystrophy
JA Hawkins-Salsbury, L Shea, X Jiang… - Journal of …, 2015 - Soc Neuroscience
Globoid cell leukodystrophy (GLD, Krabbe disease) is a lysosomal storage disease (LSD)
caused by a deficiency in galactocerebrosidase (GALC) activity. In the absence of GALC …
caused by a deficiency in galactocerebrosidase (GALC) activity. In the absence of GALC …
Preclinical studies in Krabbe disease: A model for the investigation of novel combination therapies for lysosomal storage diseases
G Heller, AM Bradbury, MS Sands, ER Bongarzone - Molecular Therapy, 2023 - cell.com
Krabbe disease (KD) is a lysosomal storage disease (LSD) caused by mutations in the galc
gene. There are over 50 monogenetic LSDs, which largely impede the normal development …
gene. There are over 50 monogenetic LSDs, which largely impede the normal development …
Extended normal life after AAVrh10-mediated gene therapy in the mouse model of Krabbe disease
MA Rafi, HZ Rao, P Luzi, MT Curtis, DA Wenger - Molecular Therapy, 2012 - cell.com
Globoid cell leukodystrophy (GLD) or Krabbe disease is a neurodegenerative disorder
caused by the deficiency of the lysosomal enzyme galactocerebrosidase (GALC). This …
caused by the deficiency of the lysosomal enzyme galactocerebrosidase (GALC). This …
Gene therapy for lysosomal storage disorders: a good start
A Biffi - Human molecular genetics, 2016 - academic.oup.com
Lysosomal storage disorders (LSDs) are a heterogeneous group of inherited diseases with
a collective frequency of∼ 1 in 7000 births, resulting from the deficiency in one or more …
a collective frequency of∼ 1 in 7000 births, resulting from the deficiency in one or more …