Combination therapies for lysosomal storage disease: is the whole greater than the sum of its parts?

Common and Uncommon Pathogenic Cascades in Lysosomal Storage Diseases

(2010) Einat B. Vitner et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Gene therapy for late infantile neuronal ceroid lipofuscinosis: neurosurgical considerations

(2010) Mark M. Souweidane et al.   Journal of Neurosurgery-Pediatrics

Oxidative stress in Niemann–Pick disease, type C

(2010) Rao Fu et al.   MOLECULAR GENETICS AND METABOLISM

Therapeutic Efficacy of Bone Marrow Transplant, Intracranial AAV-mediated Gene Therapy, or Both in the Mouse Model of MPS IIIB

(2010) Coy D Heldermon et al.   MOLECULAR THERAPY

Different Serum Enzyme Levels Are Required to Rescue the Various Systemic Features of the Mucopolysaccharidoses

(2009) Gabriella Cotugno et al.   HUMAN GENE THERAPY

Metabolic Adaptations to Interrupted Glycosaminoglycan Recycling

(2009) Josh C. Woloszynek et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Molecular signatures of disease brain endothelia provide new sites for CNS-directed enzyme therapy

(2009) Yong Hong Chen et al.   NATURE MEDICINE

Mitochondrial Ca2+ homeostasis in lysosomal storage diseases

(2008) K KISELYOV et al.   CELL CALCIUM

Treatment of Late Infantile Neuronal Ceroid Lipofuscinosis by CNS Administration of a Serotype 2 Adeno-Associated Virus Expressing CLN2 cDNA

(2008) Stefan Worgall et al.   HUMAN GENE THERAPY

The role of AMPK in psychosine mediated effects on oligodendrocytes and astrocytes: implication for Krabbe Disease

(2008) Shailendra Giri et al.   JOURNAL OF NEUROCHEMISTRY

Lentiviral-Transduced Human Mesenchymal Stem Cells Persistently Express Therapeutic Levels of Enzyme in a Xenotransplantation Model of Human Disease

(2008) Todd E. Meyerrose et al.   STEM CELLS

Biochemical, Pathological, and Skeletal Improvement of Mucopolysaccharidosis VI After Gene Transfer to Liver but Not to Muscle

(2007) Alessandra Tessitore et al.   MOLECULAR THERAPY

N-butyldeoxygalactonojirimycin reduces brain ganglioside and GM2 content in neonatal Sandhoff disease mice

(2007) Rena C. Baek et al.   NEUROCHEMISTRY INTERNATIONAL