2007

Lysosomal Storage Disorders

ISBN: 978-0-387-70908-6 (Print) 978-0-387-70909-3 (Online)

Table of contents (33 chapters)

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  1. Front Matter

    Pages i-xix

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    Book Chapter

    Pages 1-5

    From Lysosomes to Storage Diseases and Back: A Personal Reminiscence

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    Book Chapter

    Pages 7-36

    Lysosomal Biogenesis and Disease

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    Book Chapter

    Pages 37-43

    The Concept of Treatment in Lysosomal Storage Diseases

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    Book Chapter

    Pages 45-52

    Complex Lipid Catabolism

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    Book Chapter

    Pages 53-67

    Retroviral Vectors for Gene Therapy

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    Book Chapter

    Pages 69-80

    Adenovirus in Gene Therapy

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    Book Chapter

    Pages 81-95

    Setting Back the Clock: Adenoviral-Mediated Gene Therapy for Lysosomal Storage Disorders

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    Book Chapter

    Pages 97-109

    Adeno-Associated Viral-Mediated Gene Therapy of Lysosomal Storage Disorders

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    Book Chapter

    Pages 111-131

    Herpes Simplex Virus Vectors for Gene Therapy of Lysosomal Storage Disorders

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    Book Chapter

    Pages 133-151

    Gene Therapy of Lysosomal Storage Disorders by Lentiviral Vectors

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    Book Chapter

    Pages 153-168

    Substrate Reduction Therapy

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    Book Chapter

    Pages 169-178

    Newborn Screening for Lysosomal Storage Disorders

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    Book Chapter

    Pages 179-195

    Genetic Counseling for Lysosomal Storage Diseases

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    Book Chapter

    Pages 197-216

    Neural Stem Cell Therapy in Lysosomal Storage Disorders

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    Book Chapter

    Pages 217-228

    The GM1 Gangliosidoses

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    Book Chapter

    Pages 229-256

    The GM2 Gangliosidoses

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    Book Chapter

    Pages 257-268

    Acid Sphingomyelinase-Deficient Niemann–Pick Disease

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    Book Chapter

    Pages 269-283

    Krabbe Disease (Globoid Cell Leukodystrophy)

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    Book Chapter

    Pages 285-306

    Metachromatic Leukodystrophy

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    Book Chapter

    Pages 307-318

    Fabry Disease

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