Lysosomal Storage Disorders
| Corporate Author: | |
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| Other Authors: | , |
| Format: | eBook |
| Language: | English |
| Published: |
New York, NY :
Springer US : Imprint: Springer,
2007.
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| Edition: | 1st ed. 2007. |
| Subjects: | |
| Online Access: | https://doi.org/10.1007/978-0-387-70909-3 |
Table of Contents:
- 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.