iPSC are derived from skin or blood cells that have been reprogrammed back into an embryonic-like pluripotent state that enables the development of an unlimited source of any type of human cell needed for therapeutic purposes.
For example, iPSC can be prodded into becoming beta islet cells to treat diabetes, blood cells to create new blood free of cancer cells for a leukemia patient, or neurons to treat neurological disorders.
In late 2007, a BSCRC team of faculty, Drs. Kathrin Plath, William Lowry, Amander Clark, and April Pyle were among the first in the world to create human iPSC.
At that time, science had long understood that tissue specific cells, such as skin cells or blood cells, could only create other like cells. With this groundbreaking discovery, iPSC research has quickly become the foundation for a new regenerative medicine.
Using iPSC technology our faculty have reprogrammed skin cells into active motor neurons, egg and sperm precursors, liver cells, bone precursors, and blood cells.
In addition, patients with untreatable diseases such as, ALS, Rett Syndrome, Lesch-Nyhan Disease, and Duchenne’s Muscular Dystrophy donate skin cells to BSCRC scientists for iPSC reprogramming research.
The generous participation of patients and their families in this research enables BSCRC scientists to study these diseases in the laboratory in the hope of developing new treatment technologies.
The discovery in 2006 that human and mouse fibroblasts could be reprogrammed to generate induced pluripotent stem cells (iPSCs) with qualities remarkably similar to embryonic stem cells has created a valuable new source of pluripotent cells for drug discovery, cell therapy, and basic research.
Life Technologies products have been an integral part of iPSC research from the initial discovery of iPSCs to current breakthroughs. Our broad technology platform provides optimized tools for each step of the iPSC workflow: from reprogramming somatic cells to iPSC expansion, validation, and differentiation.
iPS cells and embryonic stem cells
IPS cells and embryonic stem cells are very similar. They are self-renewing, meaning they can divide and produce copies of themselves indefinitely.
Both types of stem cell can be used to derive nearly any kind of specialized cell under precisely controlled conditions in the laboratory.
Both iPS cells and embryonic stem cells can help us understand how specialized cells develop from pluripotent cells. In the future, they might also provide an unlimited supply of replacement cells and tissues for many patients with currently untreatable diseases.
In contrast to embryonic stem cells, making iPS cells doesn’t depend on the use of cells from an early embryo. Are there any other differences? Current research indicates that some genes in iPS cells behave in a different way to those in embryonic stem cells.
This is caused by incomplete reprogramming of the cells and/or genetic changes acquired by the iPS cells as they grow and multiply. Scientists are studying this in more detail to find out how such differences may affect the use of iPS cells in basic research and clinical applications.
More research is also needed to understand just how reprogramming works inside the cell. So at the moment, most scientists believe we can’t replace ES cells with iPS cells in basic research.
iPS cells and disease
An important step in developing a therapy for a given disease is understanding exactly how the disease works: what exactly goes wrong in the body? To do this, researchers need to study the cells or tissues affected by the disease, but this is not always as simple as it sounds.
For example, it’s almost impossible to obtain genuine brain cells from patients with Parkinson’s disease, especially in the early stages of the disease before the patient is aware of any symptoms.
Reprogramming means scientists can now get access to large numbers of the particular type of neurons (brain cells) that are affected by Parkinson’s disease. Researchers first make iPS cells from, for example, skin biopsies from Parkinson’s patients.
They then use these iPS cells to produce neurons in the laboratory. The neurons have the same genetic background (the same basic genetic make-up) as the patients’ own cells. Thus scientist can directly work with neurons affected by Parkinson’s disease in a dish.
They can use these cells to learn more about what goes wrong inside the cells and why. Cellular ‘disease models’ like these can also be used to search for and test new drugs to treat or protect patients against the disease.
Future applications and challenges for iPS cells
Reprogramming holds great potential for new medical applications, such as cell replacement therapies. Since iPS cells can be made from a patient’s own skin, they could be used to grow specialized cells that exactly match the patient and would not be rejected by the immune system.
If the patient has a genetic disease, the genetic problem could be corrected in their iPS cells in the laboratory, and these repaired iPS cells used to produce a patient-specific batch of healthy specialized cells for transplantation. But this benefit remains theoretical for now.
Until recently, making iPS cells involved permanent genetic changes inside the cell, which can cause tumours to form. Scientists have now developed methods for making iPS cells without this genetic modification.
These new techniques are an important step towards making iPS-derived specialized cells that would be safe for use in patients.
Further research is now needed to understand fully how reprogramming works and how iPS cells can be controlled and produced consistently enough to meet the high quality and safety requirements for use in the clinic.