olga ujhelly, phd during my science in biotalentum, gödöllő, hungary 25-29.1
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Induced Pluripotent Stem (iPS) cells - as new tools for future
regenerative medicine and drug testing
Dr. Olga Ujhelly
BioTalentum Ltd., Hungary
Pluripotent stem cells have the capacity to become any cell
in the body
Multiple differentiation potential = PLURIPOTENT
Cardiac muscle
Skeletal muscle
Kidney tubule cell
Pigment cell
Neuron cell
Skin cell
Pancreatic cell
Lung cell
Thyroid cell
Smooth muscle
Red blood cells
Differentiation
Pluripotent
stem cells
Somatic cells
Types of pluripotent stem cells
Embrionic stem cells - derived from blastocyst
Nuclear transfer-Embrionic stem cells - derived by somatic cell nuclear
Require embryos or eggs to create
Induced pluripotent stem cell
New technology, developed by Shinya Yamanaka in 2006
Derived from somatic cells without sacrificing embryos
Method of the Year 2009: iPS cells
Generarion of induced pluripotent stem cells
In plutipotent stem cell a set of genes are active, which are required for maintaining
plripotency
In somatic cells most of these genes are inactive
Some genes required for
pluripotency:
FoxD3
FbxoERAS
Nanog
Zfp296
Rex1
Dax1
Sox2
Oct4
Klf4
c-myc
Generarion of induced pluripotent stem cells
In plutipotent stem cell a set of genes are active, which are required for maintaining
plripotency
In somatic cells most of these genes are inactive
Some genes required for
pluripotency:
FoxD3
FbxoERAS
Nanog
Zfp296
Rex1
Dax1
Sox2
Oct4
Klf4
c-myc
Expression four of these genes in somatic cells induces pluripotency
Somatic cells iPS cellSox2
Oct4 Klf4
c-myc
2 weeks
Pluripotent stem cells can be generated from somatic cells
Multiple differentiation potential = PLURIPOTENT
Pluripotent
stem cells
Cardiac muscle
Skeletal muscle
Kidney tubule cell
Pigment cell
Neuron cell
Skin cell
Pancreatic cell
Lung cell
Thyroid cell
Smooth muscle
Red blood cells
Differentiation
De-Differentiation
Somatic cells
The characteristics of iPS cells are very similar to ES cells
• Morphology
• SSEA1 and alkaline phosphatase staining
• In vitro differentiation
EB formation
• In vivo differentiation
Teratoma formation
In vivo pluripotency: chimeras
iPS cells were generated from different species
2006 - Mouse
2007 - Human
2008 - Monkey
2009 - Rat
2009 - Pig
2009 - Dog
Potential applications of iPS cells
Therapy
Conservation research
Basic research
Pharmaceutical studies
Patient specific pluripotent stem cells, which can be differentiated to all the cell
types of the body
Cell replacement therapy for cardiac diseases, diabetes, parkinson disease etc.
Studying signaling pathways involved in pluripotency
Studying early embryonic development
Studying development of different species
Generation models for human diseases
Generation iPS cells from endangered species
Gene therapy - e.g. sickle cell anemia
Generation cell lines for drug testing
iPS cellSox2
Oct4 Klf4
c-myc
De-differentiation
Differentiation
Somatic cells
Cell therapy - regenerative medicine
iPS cellSox2
Oct4 Klf4
c-myc
De-differentiation
Somatic cells
Gene therapy
Sickle cell anemia
Hemoglobin gene
mutation
Hemoglobin gene
modification
Gene-modified iPS cell
Differentiation
Hematopoietic
stem cells
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