The knowledge of lysosomal biology and the consequences of its dysfunction have increased dramatically in the past 60 years. This book describes the nature of the lysosomal dysfunction and diseases as well as potential future treatments and therapies. Disease specific chapters provide thorough reviews of the clinical features of lysosomal storage disorders, their molecular basis and the commercial or experimental therapeutic approaches sought in this area. This is an invaluable resource for researchers in biochemical and molecular genetics, enzyme therapy, and gene transfer.

The knowledge of lysosomal biology and the consequences of its dysfunction have increased dramatically in the past 60 years. Research of these disorders has moved from diseases with unknown etiology to disorders with clear and defined pathophysiology and some of them have benefited from the development of disease specific therapeutics. This book describes the nature of the lysosomal dysfunction and diseases as well as potential future treatments and therapies. Disease specific chapters provide thorough reviews of the clinical features of lysosomal storage disorders, their molecular basis and the commercial or experimental therapeutic approaches sought in this area. This is an invaluable resource for researchers in biochemical and molecular genetics, enzyme therapy, and gene transfer.

From Lysosomes to Storage Diseases and Back: A Personal Reminiscence.- Lysosomal Biogenesis and Disease.- The 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 Lysomal 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.- The GM1 Gangliosidoses.- The 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.- The Neuronal Ceroid Lipofuscinoses: Clinical Features and Molecular Basis of Disease.- Mucopolysaccharidosis I.- Mucopolysaccharidosis II.- Sanfilippo Syndrome: Clinical Genetic Diagnosis and Therapies.- Mucopolysaccharidosis IV (Morquio Syndrome; MPS IV).- Mucopolysaccharidosis Type VI (MPS 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.