Lysosomal Disorders of the Brain
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Lysosomal Disorders of the Brain
Recent Advances in Molecular and Cellular Pathogenesis and Treatment
Platt, Frances; Walkley, Steven
Oxford University Press
02/2004
478
Dura
Inglês
9780198508786
15 a 20 dias
1079
Descrição não disponível.
Foreword ; Preface ; Prologue ; SECTION I: OVERVIEW OF LYSOSOMES AND STORAGE DISEASES ; 1. The endosomal-lysosomal system ; 2. Lysosomal defects and storage ; 3. Clinical aspects and diagnosis ; SECTION II: MOLECULAR MECHANISMS OF STORAGE ; 4. Primary defects in lysosomal enzymes ; 5. Defects in lysosomal enzyme modification for catalytic activity ; 6. Defects in lysosomal enzyme trafficking ; 7. Defects in lysosomal enzyme protection: galactosialidosis ; 8. Defects in activator proteins and other soluble proteins of the lysosome ; 9. Defects in transmembrane proteins ; SECTION III: MODEL SYSTEMS AND PATHOPHYSIOLOGICAL MECHANISMS ; 10. Simple non-mammalian systems ; 11. Spontaneous and engineered mammalian storage disease models ; 12. Pathogenic cascades and brain dysfunction ; SECTION IV: TREATMENT OF STORAGE DISEASES ; 13. Enzyme replacement therapy ; 14. Cell-mediated delivery systems ; 15. Inhibition of substrate synthesis: a pharmacological approach for glycosphingolipid storage disease therapy ; 16. Gene therapy
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Foreword ; Preface ; Prologue ; SECTION I: OVERVIEW OF LYSOSOMES AND STORAGE DISEASES ; 1. The endosomal-lysosomal system ; 2. Lysosomal defects and storage ; 3. Clinical aspects and diagnosis ; SECTION II: MOLECULAR MECHANISMS OF STORAGE ; 4. Primary defects in lysosomal enzymes ; 5. Defects in lysosomal enzyme modification for catalytic activity ; 6. Defects in lysosomal enzyme trafficking ; 7. Defects in lysosomal enzyme protection: galactosialidosis ; 8. Defects in activator proteins and other soluble proteins of the lysosome ; 9. Defects in transmembrane proteins ; SECTION III: MODEL SYSTEMS AND PATHOPHYSIOLOGICAL MECHANISMS ; 10. Simple non-mammalian systems ; 11. Spontaneous and engineered mammalian storage disease models ; 12. Pathogenic cascades and brain dysfunction ; SECTION IV: TREATMENT OF STORAGE DISEASES ; 13. Enzyme replacement therapy ; 14. Cell-mediated delivery systems ; 15. Inhibition of substrate synthesis: a pharmacological approach for glycosphingolipid storage disease therapy ; 16. Gene therapy
Este título pertence ao(s) assunto(s) indicados(s). Para ver outros títulos clique no assunto desejado.
