Treatment for Krabbe's disease: Finding the combination

Lysosomal Storage Diseases: From Pathophysiology to Therapy

(2015) Giancarlo Parenti et al.   Annual Review of Medicine

Mechanism-Based Combination Treatment Dramatically Increases Therapeutic Efficacy in Murine Globoid Cell Leukodystrophy

(2015) J. A. Hawkins-Salsbury et al.   JOURNAL OF NEUROSCIENCE

Intravenous injection of AAVrh10-GALC after the neonatal period in twitcher mice results in significant expression in the central and peripheral nervous systems and improvement of clinical features

(2015) Mohammad A. Rafi et al.   MOLECULAR GENETICS AND METABOLISM

Long-term Improvements in Lifespan and Pathology in CNS and PNS After BMT Plus One Intravenous Injection of AAVrh10-GALC in Twitcher Mice

(2015) Mohammad A Rafi et al.   MOLECULAR THERAPY

Transplantation of mouse embryonic stem cell-derived oligodendrocytes in the murine model of globoid cell leukodystrophy

(2015) Xiao Ling Kuai et al.   Stem Cell Research & Therapy

Effect of vitamin D3 intake on the onset of disease in a murine model of human Krabbe disease

(2014) Manjeet K. Paintlia et al.   JOURNAL OF NEUROSCIENCE RESEARCH

Early axonal loss accompanied by impaired endocytosis, abnormal axonal transport, and decreased microtubule stability occur in the model of Krabbe's disease

(2014) Carla Andreia Teixeira et al.   NEUROBIOLOGY OF DISEASE

Role of endogenous psychosine accumulation in oligodendrocyte differentiation and survival: Implication for Krabbe disease

(2013) Je-Seong Won et al.   BRAIN RESEARCH

Missense mutation in mouse GALC mimics human gene defect and offers new insights into Krabbe disease

(2013) Gregory B. Potter et al.   HUMAN MOLECULAR GENETICS

Psychosine, the cytotoxic sphingolipid that accumulates in globoid cell leukodystrophy, alters membrane architecture

(2013) Jacqueline A. Hawkins-Salsbury et al.   JOURNAL OF LIPID RESEARCH

Enzyme Replacement Therapy for Lysosomal Diseases: Lessons from 20 Years of Experience and Remaining Challenges

(2012) R.J. Desnick et al.   Annual Review of Genomics and Human Genetics

Oxidative stress as a therapeutic target in globoid cell leukodystrophy

(2012) Jacqueline A. Hawkins-Salsbury et al.   EXPERIMENTAL NEUROLOGY

Bone marrow transplantation increases efficacy of central nervous system-directed enzyme replacement therapy in the murine model of globoid cell leukodystrophy

(2012) Elizabeth Y. Qin et al.   MOLECULAR GENETICS AND METABOLISM

Extended Normal Life After AAVrh10-mediated Gene Therapy in the Mouse Model of Krabbe Disease

(2012) Mohammad A Rafi et al.   MOLECULAR THERAPY

The AAV Vector Toolkit: Poised at the Clinical Crossroads

(2012) Aravind Asokan et al.   MOLECULAR THERAPY

Early Infantile Krabbe Disease: Results of the World-Wide Krabbe Registry

(2012) Patricia K. Duffner et al.   PEDIATRIC NEUROLOGY

Axonopathy is a compounding factor in the pathogenesis of Krabbe disease

(2011) Ludovico Cantuti Castelvetri et al.   ACTA NEUROPATHOLOGICA

Effect of intrastriatal mesenchymal stromal cell injection on progression of a murine model of Krabbe disease

(2011) Shawna E. Wicks et al.   BEHAVIOURAL BRAIN RESEARCH

Bone Marrow Transplantation Augments the Effect of Brain- and Spinal Cord-Directed Adeno-Associated Virus 2/5 Gene Therapy by Altering Inflammation in the Murine Model of Globoid-Cell Leukodystrophy

(2011) A. S. Reddy et al.   JOURNAL OF NEUROSCIENCE

Insights into Krabbe disease from structures of galactocerebrosidase

(2011) J. E. Deane et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Systemic Delivery of Bone Marrow-Derived Mesenchymal Stromal Cells Diminishes Neuropathology in a Mouse Model of Krabbe's Disease

(2011) Catarina Oliveira Miranda et al.   STEM CELLS

Krabbe leukodystrophy in a selected population with high rate of late onset forms: longer survival linked to c.121G>A (p.Gly41Ser) mutation

(2010) A Fiumara et al.   CLINICAL GENETICS

Identification and characterization of 15 novel GALC gene mutations causing Krabbe disease

(2010) Barbara Tappino et al.   HUMAN MUTATION

Identification of Hematopoietic Stem Cell-Specific miRNAs Enables Gene Therapy of Globoid Cell Leukodystrophy

(2010) B. Gentner et al.   Science Translational Medicine

Mesenchymal Lineage Stem Cells Have Pronounced Anti-Inflammatory Effects in the Twitcher Mouse Model of Krabbe's Disease

(2010) Cynthia B. Ripoll et al.   STEM CELLS

Effects of treatments on inflammatory and apoptotic markers in the CNS of mice with globoid cell leukodystrophy

(2009) Paola Luzi et al.   BRAIN RESEARCH

Psychosine Accumulates in Membrane Microdomains in the Brain of Krabbe Patients, Disrupting the Raft Architecture

(2009) A. B. White et al.   JOURNAL OF NEUROSCIENCE

Combined hematopoietic and lentiviral gene-transfer therapies in newborn Twitcher mice reveal contemporaneous neurodegeneration and demyelination in Krabbe disease

(2009) F. Galbiati et al.   JOURNAL OF NEUROSCIENCE RESEARCH

Significant correction of pathology in brains of twitcher mice following injection of genetically modified mouse neural progenitor cells

(2009) Michelina Strazza et al.   MOLECULAR GENETICS AND METABOLISM