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Lysosomal Storage Disorders

John A. Barranger and Mario A. Cabrera-Salazar

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Contents
ESM

i-xix
Front matter
1-5
From Lysosomes to Storage Diseases and Back: A Personal Reminiscence
7-36
Lysosomal Biogenesis and Disease
37-43
The Concept of Treatment in Lysosomal Storage Diseases
45-52
Complex Lipid Catabolism
53-67
Retroviral Vectors for Gene Therapy
69-80
Adenovirus in Gene Therapy
81-95
Setting Back the Clock: Adenoviral-Mediated Gene Therapy for Lysosomal Storage Disorders
97-109
Adeno-Associated Viral-Mediated Gene Therapy of Lysosomal Storage Disorders
111-131
Herpes Simplex Virus Vectors for Gene Therapy of Lysosomal Storage Disorders
133-151
Gene Therapy of Lysosomal Storage Disorders by Lentiviral Vectors
153-168
Substrate Reduction Therapy
169-178
Newborn Screening for Lysosomal Storage Disorders
179-195
Genetic Counseling for Lysosomal Storage Diseases
197-216
Neural Stem Cell Therapy in Lysosomal Storage Disorders
217-228
The GM1 Gangliosidoses
229-256
The GM2 Gangliosidoses
257-268
Acid Sphingomyelinase-Deficient Niemann–Pick Disease
269-283
Krabbe Disease (Globoid Cell Leukodystrophy)
285-306
Metachromatic Leukodystrophy
307-318
Fabry Disease
319-343
Gaucher Disease: Review and Perspectives on Treatment
345-370
Therapeutic Goals in the Treatment of Gaucher Disease
371-388
The Neuronal Ceroid Lipofuscinoses: Clinical Features and Molecular Basis of Disease
389-405
Mucopolysaccharidosis I
407-414
Mucopolysaccharidosis II (Hunter Syndrome)
415-432
Sanfilippo Syndrome: Clinical Genetic Diagnosis and Therapies
433-445
Mucopolysaccharidosis IV (Morquio Syndrome)
447-456
Mucopolysaccharidosis Type VI (Maroteaux–Lamy Syndrome)
457-472
Mucopolysaccharidosis Type VII (Sly Disease): Clinical, Genetic Diagnosis and Therapies
473-498
Pompe Disease-Glycogenosis Type II: Acid Maltase Deficiency
499-511
Lysosomal Free Sialic Acid Storage Disorders: Salla Disease and ISSD
513-527
Cystinosis
529-537
I-Cell Disease
539-562
Back matter

About This Book

Title

Lysosomal Storage Disorders

Authors

Author Affiliations

Department of Human Genetics, University of Pittsburgh, 15261 Pittsburgh, PA USA
Genzyme Corporation, 01701–9322, Framingham, MA USA

Collection

Biomedical and Life Sciences

Subjects

Copyright Year

2007

DOI

10.1007/978-0-387-70909-3

ISBN

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

Additional Links

About This Book

Publisher

Springer US

SpringerLink Date

Tuesday, October 16, 2007

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