A new therapeutic approach: take a few epithelial (skin) cells or blood cells from a patient and transform them to stem cells that can then be developed to any cell type for research into mechanism of patient’s disease.
An example from Harvard on the occasion of a recent live event
Kevin Eggan, Ph.D. Professor in the Department of Stem Cell and Regenerative Biology, Harvard University http://hsci.harvard.edu/people/kevin-c-eggan-phd
Excerpt: “While training, Dr. Eggan performed nuclear transfer studies that challenged preconceived notions concerning the limits of cellular plasticity (Eggan et al., 2000, 2001, 2004). His own lab then became the first to demonstrate that human somatic cells could be reprogrammed to an embryonic stem (ES) cell state (Cowan et al., 2005).”
Amylotrophic Lateral Sclerosis or ALS (the disease of Dr. Stephen Hawkings) results in degeneration of motor neurons i.e. the neurons that control voluntary muscles. If you have a cell in a dish with this disease, how do you determine what is wrong with it?
Excerpt: “In 2008, Professor Eggan first raised the possibility of growing ALS patient-derived stem cells in a laboratory dish. At that time, there was no way to directly access and study living human tissue with the disease. Patient-grown stem cells have since allowed scientists to explore how specific mutations cause the disease and screen for drugs that might fix these problems. This year (2014), the Eggan Lab reported two major findings as a result of their work. (…)”
When you have a neuron in a dish, you want to study its electrical behavior, how it generates electricity and how electricity is propagated in the network. This is a task for Adam E. Cohen, Professor of Chemistry and Chemical Biology and of Physics, Harvard University https://www.physics.harvard.edu/people/facpages/cohen
Excerpts: “Cohen developed a device that uses light-sensitive proteins to stimulate and record electrical energy in a single cell and in networks of cells.
Using this tool, Eggan and colleague Clifford Woolf measured the activity of ALS-diseased motor neurons. ALS is a neurodegenerative disease that attacks the circuit that sends electrical impulses from the brain, down long motor neurons extending out into the limbs, and into the muscle tissue. (…)”
“Eggan and Woolf found that the diseased motor neurons are hyperexcitable, meaning they fire more often than healthy neurons. Because they are more active, the diseased motor neurons wear out more quickly and expire, severing the line of communication between the brain and the muscles. (…)”
“The scientists used the ALS-affected motor neurons to test for compounds that calm the hyperexcitability and found that an FDA-approved drug called retigabine may be a potential therapeutic.”
Note: A clinical trial is currently in progress.
At a Harvard live event on 2016-10-18, Adam E. Cohen demonstrated how it is possible to stimulate one neuron and follow how the signal hops in the network and thereby how information is propagated. Please refer to the following picture (cell that is stimulated marked with a blue dot, indicated by arrow) and the demonstration that starts at time point 9.00 of the video