Lysosomal Storage Disorders

From lysosomes to storage diseases and back: a personal reminiscence -- Lysosomal biogenesis and disease -- Concept of treatment in lysosomal storage diseases -- Complex lipid catabolism -- Retroviral vectors for gene therapy -- Adenovirus in gene therapy -- Setting back the clock: adenoviral-mediated gene therapy for lysosomal storage disorders -- Adeno-associated viral-mediated gene therapy of lysosomal storage disorders -- Herpes simplex virus vectors for gene therapy of lysosomal storage disorders -- Gene therapy of lysosomal storage disorders by lentiviral vectors -- Substrate reduction therapy -- Newborn screening for lysosomal storage disorders -- Genetic counseling for lysosomal storage diseases -- Neural stem cell therapy in lysosomal storage disorders -- GM1 gangliosidoses -- GM2 gangliosidoses -- Acid sphingomyelinase-deficient Niemann-Pick disease -- Krabbe disease (globoid cell leukodystrophy) -- Metachromatic leukodystrophy -- Fabry disease -- Gaucher disease: review and perspectives on treatment -- Therapeutic goals in the treatment of gaucher disease -- Neuronal ceroid lipofuscinoses: clinical features and molecular basis of disease -- Mucopolysaccharidosis I -- Mucopolysaccharidosis II (Hunter syndrome) -- Sanfilippo syndrome: clinical genetic diagnosis and therapies -- Mucopolysaccharidosis IV (Morquio syndrome) -- Mucopolysaccharidosis type VI (Maroteaux-Lamy syndrome) -- Mucopolysaccharidosis type VII (Sly disease): clinical, genetic diagnosis and therapies -- Pompe disease-glycogenosis type II: acid maltase deficiency -- Lysosomal free sialic acid storage disorders: Salla disease and ISSD -- Cystinosis -- I-cell disease.