Lysosomal Storage Disorders - Barranger, John A.; Cabrera-Salazar, Mario; SpringerLink (Online service) (New York, NY : Springer US : Imprint: Springer, 2007)
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 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 -- 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 (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.
Keywords: Cell biology; Human genetics; Epidemiology; Biochemistry; Cell Biology; Human Genetics; Epidemiology; Biochemistry, general