Recent Advances in Hematopoietic Stem Cell Gene Therapy ( Innovations in Stem Cell Transplantation )

Publication series ： Innovations in Stem Cell Transplantation

Author： Toshihisa Tsuruta  

Publisher： IntechOpen‎

Publication year： 2013

E-ISBN: INT5608253587

P-ISBN(Paperback): 9789535109808

P-ISBN(Hardback):  9789535109808

Subject： Q2 Cytobiology

Keyword： 细胞生物学

Language： ENG

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Recent Advances in Hematopoietic Stem Cell Gene Therapy

Chapter

Chapter 5 Recent Advances in Hematopoietic Stem Cell Gene Therapy

1. Introduction

2. Characteristics of HSCs and gene therapy

3. Vectors for HSC gene therapy

3.1. Gammaretroviral (Retroviral) vectors

3.2. Lentiviral vectors

3.3. Genotoxicity of viral vectors

4. Clinical applications of HSC gene therapy

4.1. β-thalassemia

4.2. Fanconi anemia

4.3. Hemophilia

4.4. Primary immunodeficiencies

4.4.1. SCID-X1

4.4.2. ADA-SCID

4.4.3. CGD

4.4.4. Wiskott-Aldrich Syndrome (WAS)

4.4.5. Janus Kinase 3 (JAK3) deficiency

4.4.6. Purine Nucleoside Phosphorylase (PNP) deficiency

4.4.7. Leukocyte Adhesion Deficiency type 1 (LAD-1)

4.5. Mucopolysaccharidosis (MPS)

4.6. Gaucher disease

4.7. X-ALD

5. Conclusion

Author details

References

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