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

description

Second team of 15 young media makers had a meeting in Gödöllő, Hungary for The Stem Cell and Nuclear Transfer Cloning technologies workshop at BioTalentum. This was the second meeting in the framework of My Science Program. Study visit started on 25th and last till 29th January 2010.

Transcript of Olga Ujhelly, PhD during My Science in Biotalentum, Gödöllő, Hungary 25-29.1

Page 1: Olga Ujhelly, PhD during My Science in Biotalentum, Gödöllő, Hungary 25-29.1

Induced Pluripotent Stem (iPS) cells - as new tools for future

regenerative medicine and drug testing

Dr. Olga Ujhelly

BioTalentum Ltd., Hungary

Page 2: Olga Ujhelly, PhD during My Science in Biotalentum, Gödöllő, Hungary 25-29.1

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

Page 3: Olga Ujhelly, PhD during My Science in Biotalentum, Gödöllő, Hungary 25-29.1

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

Page 4: Olga Ujhelly, PhD during My Science in Biotalentum, Gödöllő, Hungary 25-29.1

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

Page 5: Olga Ujhelly, PhD during My Science in Biotalentum, Gödöllő, Hungary 25-29.1

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

Page 6: Olga Ujhelly, PhD during My Science in Biotalentum, Gödöllő, Hungary 25-29.1

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

Page 7: Olga Ujhelly, PhD during My Science in Biotalentum, Gödöllő, Hungary 25-29.1

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

Page 8: Olga Ujhelly, PhD during My Science in Biotalentum, Gödöllő, Hungary 25-29.1

In vivo pluripotency: chimeras

Page 9: Olga Ujhelly, PhD during My Science in Biotalentum, Gödöllő, Hungary 25-29.1

iPS cells were generated from different species

2006 - Mouse

2007 - Human

2008 - Monkey

2009 - Rat

2009 - Pig

2009 - Dog

Page 10: Olga Ujhelly, PhD during My Science in Biotalentum, Gödöllő, Hungary 25-29.1

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

Page 11: Olga Ujhelly, PhD during My Science in Biotalentum, Gödöllő, Hungary 25-29.1

iPS cellSox2

Oct4 Klf4

c-myc

De-differentiation

Differentiation

Somatic cells

Cell therapy - regenerative medicine

Page 12: Olga Ujhelly, PhD during My Science in Biotalentum, Gödöllő, Hungary 25-29.1

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