Reversibility of neuropathology in Tay–Sachs-related diseases

Disease correction by combined neonatal intracranial AAV and systemic lentiviral gene therapy in Sanfilippo Syndrome type B mice

(2013) C D Heldermon et al.   GENE THERAPY

Therapeutic Response in Feline Sandhoff Disease Despite Immunity to Intracranial Gene Therapy

(2013) Allison M Bradbury et al.   MOLECULAR THERAPY

Gene Transfer Corrects Acute GM2 Gangliosidosis—Potential Therapeutic Contribution of Perivascular Enzyme Flow

(2012) M Begoña Cachón-González et al.   MOLECULAR THERAPY

Characterization of Inducible Models of Tay-Sachs and Related Disease

(2012) Timothy J. Sargeant et al.   PLoS Genetics

Myelin genes are downregulated in canine fucosidosis

(2011) Jessica L. Fletcher et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE

Adeno-associated virus-mediated expression of β-hexosaminidase prevents neuronal loss in the Sandhoff mouse brain

(2011) Timothy J. Sargeant et al.   HUMAN MOLECULAR GENETICS

Galactolipid deficiency in the early pathogenesis of neuronal ceroid lipofuscinosis model Cln8mnd: implications to delayed myelination and oligodendrocyte maturation

(2011) M. Kuronen et al.   NEUROPATHOLOGY AND APPLIED NEUROBIOLOGY

Iminosugar-Based Inhibitors of Glucosylceramide Synthase Increase Brain Glycosphingolipids and Survival in a Mouse Model of Sandhoff Disease

(2011) Karen M. Ashe et al.   PLoS One

Autophagy gone awry in neurodegenerative diseases

(2010) Esther Wong et al.   NATURE NEUROSCIENCE

Pathology of GM2 Gangliosidosis in Jacob Sheep

(2010) B. F. Porter et al.   VETERINARY PATHOLOGY

Alterations of myelin-specific proteins and sphingolipids characterize the brains of acid sphingomyelinase-deficient mice, an animal model of Niemann-Pick disease type A

(2009) Barbara Buccinnà et al.   JOURNAL OF NEUROCHEMISTRY

Early changes in the apparent diffusion coefficient (ADC) in a mouse model of Sandhoff's disease occur prior to disease symptoms and behavioral deficits

(2009) Lingyun Hu et al.   MAGNETIC RESONANCE IN MEDICINE

Early deficits in motor coordination and cognitive dysfunction in a mouse model of the neurodegenerative lysosomal storage disorder, Sandhoff disease

(2008) Maria Gulinello et al.   BEHAVIOURAL BRAIN RESEARCH

Comparative Analysis of Brain Lipids in Mice, Cats, and Humans with Sandhoff Disease

(2008) Rena C. Baek et al.   LIPIDS