Barranger, John A.
Lysosomal Storage Disorders
1. From Lysosomes to Storage Diseases and Back: A Personal Reminiscence
Christian Duve
2. Lysosomal Biogenesis and Disease
Doug Brooks, Emma Parkinson-Lawrence
3. The Concept of Treatment in Lysosomal Storage Diseases
Roscoe O. Brady
4. Complex Lipid Catabolism
Roscoe O. Brady, Roscoe O. Brady
5. Retroviral Vectors for Gene Therapy
Seon-Hee Kim, Paul D. Robbins
6. Adenovirus in Gene Therapy
Angela Montecalvo, Andrea Gambotto, Leonardo D’Aiuto
7. Setting Back the Clock: Adenoviral-Mediated Gene Therapy for Lysosomal Storage Disorders
Dolan Sondhi, Neil R. Hackett, Stephen M. Kaminksy, Ronald G. Crystal
8. Adeno-Associated Viral-Mediated Gene Therapy of Lysosomal Storage Disorders
Mario A. Cabrera-Salazar, Seng H. Cheng
9. Herpes Simplex Virus Vectors for Gene Therapy of Lysosomal Storage Disorders
Edward A. Burton, Joseph C. Glorioso
10. Gene Therapy of Lysosomal Storage Disorders by Lentiviral Vectors
Alessandra Biffi, Luigi Naldini
11. Substrate Reduction Therapy
Frances M. Platt, Terry D. Butters
12. Newborn Screening for Lysosomal Storage Disorders
C. Ronald Scott, Frantisek Turecek, Michael H. Gelb
13. Genetic Counseling for Lysosomal Storage Diseases
Erin O'Rourke, Dawn Laney, Cindy Morgan, Kim Mooney, Jennifer Sullivan
14. Neural Stem Cell Therapy in Lysosomal Storage Disorders
Jean-Pyo Lee, Dan Clark, Mylvaganam Jeyakumar, Rodolfo Gonzalez, Scott Mckercher, Franz-Josef Muller, Rahul Jandial, Rosanne M. Taylor, Kook Park, Thomas N. Seyfried, Frances M. Platt, Evan Y. Snyder
15. The GM1 Gangliosidoses
Gustavo Charria-Ortiz
16. The GM2 Gangliosidoses
Gustavo A. Charria-Ortiz
17. Acid Sphingomyelinase-Deficient Niemann–Pick Disease
Edward H. Schuchmann, Margaret McGovern, Calogera M. Simonaro, Melissa P. Wasserstein, Robert J. Desnick
18. Krabbe Disease (Globoid Cell Leukodystrophy)
Junko Matsuda, Kunihiko Suzuki
19. Metachromatic Leukodystrophy
Volkmar Gieselmann
20. Fabry Disease
Roscoe O. Brady
21. Gaucher Disease: Review and Perspectives on Treatment
Mario A. Cabrera-Salazar, John A. Barranger
22. Therapeutic Goals in the Treatment of Gaucher Disease
Neal Weinreb
23. The Neuronal Ceroid Lipofuscinoses: Clinical Features and Molecular Basis of Disease
Beverly L. Davidson, Mario A. Cabrera-Salazar, David A. Pearce
24. Mucopolysaccharidosis I
Lorne A. Clarke
25. Mucopolysaccharidosis II (Hunter Syndrome)
Lorne A. Clarke
26. Sanfilippo Syndrome: Clinical Genetic Diagnosis and Therapies
John J. Hopwood
27. Mucopolysaccharidosis IV (Morquio Syndrome)
Shunji Tomatsu, Adriana M. Montaño, Tatsuo Nishioka, Tadao Orii
28. Mucopolysaccharidosis Type VI (Maroteaux–Lamy Syndrome)
James. E. Wraith
29. Mucopolysaccharidosis Type VII (Sly Disease): Clinical, Genetic Diagnosis and Therapies
Denise J. Norato
30. Pompe Disease-Glycogenosis Type II: Acid Maltase Deficiency
Arnold Reuser, Marian Kroos
31. Lysosomal Free Sialic Acid Storage Disorders: Salla Disease and ISSD
Amanda Helip-Wooley, Robert Kleta, William A. Gahl
32. Cystinosis
Amanda Helip-Wooley, Robert Kleta, William A. Gahl
33. I-Cell Disease
Doug Brooks, Chris Turner, Viv Muller, John J. Hopwood, Peter Meikle
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- Författare
- Barranger, John A.
- Cabrera-Salazar, Mario A.
- Utgivare
- Springer
- Utgivningsår
- 2008
- Språk
- en
- Utgåva
- 1
- Kategori
- Hälsa, skönhet, mode
- Format
- E-bok
- eISBN (PDF)
- 9780387709093