Molecular Therapy for Lysosomal Storage Diseases ( Gene Therapy - Tools and Potential Applications )

Publication series ： Gene Therapy - Tools and Potential Applications

Author： Daisuke Tsuji and Kohji Itoh  

Publisher： IntechOpen‎

Publication year： 2013

E-ISBN: INT5019454074

P-ISBN(Paperback): 9789535110149

P-ISBN(Hardback):  9789535110149

Subject： R394 medical genetics

Keyword： 医学遗传学

Language： ENG

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Molecular Therapy for Lysosomal Storage Diseases

Chapter

Chapter 24 Molecular Therapy for Lysosomal Storage Diseases

1. Introduction

2. GM2 gangliosidoses

3. General aspects of gene therapy for LSDs

4. Gene therapy for GM2 gangliosidoses

4.1. Experimental and preclinical gene therapy using animal models

4.2. CNS-directed in vivo gene therapy

4.3. CNS-directed ex vivo gene therapy

5. Conclusion

Author details

References

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