This document discusses stem cell gene therapy. It defines stem cells as cells that can develop into different types of cells and have the ability to continuously divide. There are two main types of stem cells: embryonic stem cells which are highly undifferentiated and adult stem cells which help maintain and repair tissues. Stem cell gene therapy involves using stem cells and transfecting or transducing them to introduce new genes to treat diseases like hemophilia, heart disease, and sickle cell disease. The advantages are potential cures for many diseases, while the disadvantages include additional biological complexity and possible negative effects on neighboring genes.

2. Contents
 General introduction
 Gene therapy
 Stem cell gene therapy
 Applications
 Advantages & Disadvantages

3. Stem Cell Gene Therapy
General Introduction
Stem cells

4. Stem cells
 Stem cells have the remarkable potential to develop
into many different cell types in the body during
early life and growth
 Pluripotent cells

5. Stem cells
 Ability to divide numerous times and produce
progeny that undergo differentiation
1
Differentiation =
division power

6. Types of stem cells
Two types of stem cells:

Embryonic Stem Cells
 Highly undifferentiated
 Allogeneic transplantation
 Taken from umbilical cord

7. Adult Stem Cells
 Somatic stem cell
 Maintain and repair the tissue
 Autologous transplantation

8. Gene Therapy (GT)
Stem Cells Gene Therapy
 Use of adult stem cell or embryonic stem cell
 Transfection
 Transduction

9. Stem Cells Gene Therapy

10. Applications of stem cell gene therapy
 Hemophilia treatment
 Repairing mutations
 Heart disease treatment
 Sickle Cell Disease treatment
 Skin disease treatment

11. Stem Cell Gene Therapy for Sickle Cell
Disease
 Sickle cell disease (SCD) is common monogenic
disorders worldwide
 Major cause of early mortality
 Inherited abnormality of the oxygen-carrying
hemoglobin protein in red blood cells (RBC)

13.  Source of RBCs is bone marrow

14. Advantages
 It discover treatments and cures to diseases like
Cancer, spinal cord injuries, diabetes & many
more
 It provides medical benefits in the fields of
therapeutic cloning
 Healthy life

15. Disadvantage
 Additional biological complexity brought into
systems by living cells
 Some times it leads to mortality of individual
 Modify the activity of neighboring genes
(positively or negatively)

16. References
 Thorat, C. et al. What the Orphan Drug Act has done lately for
children with rare diseases: a 10-year analysis. Pediatrics 129,
516–521 (2012). | Article | PubMed |
 Grieger, J.C. & Samulski, R.J. Adeno-associated virus
vectorology, manufacturing, and clinical applications. Meth.
Enzymol. 507, 229–254 (2012). | PubMed |
 http://stemcells.nih.gov/info/scireport/pages/chapter11.aspx
 http://www.explorestemcells.co.uk/StemCellsAndGeneTherap
y.html