Forum for Science, Industry and Business

Sponsored by:     3M 
Search our Site:

 

Stem cells move one step closer to cure for genetic diseases

16.07.2015

Healthy brain, muscle, eye and heart cells would improve the lives of tens of thousands of people around the world with debilitating mitochondrial diseases. Now, researchers at the Salk Institute have gotten one step closer to making such cures a reality: they've turned cells from patients into healthy, mutation-free stem cells that can then become any cell type. The new approach is described July 15, 2015 in Nature.

"Right now, there are no cures for mitochondrial diseases," says senior author Juan Carlos Izpisua Belmonte, professor in Salk's Gene Expression Laboratory. "Very recently, we've developed ways to prevent these diseases, so it was natural to next ask how we could treat them."


Salk researchers have generated disease-free stem cells from patients with mitochondrial disease that can be converted into any cell type including neuronal progenitors (left) or heart cells (right). These could potentially be used for future transplantation into patients.

Credit: Salk Institute

Mitochondrial diseases are caused by any of about 200 mutations that affect the genes of mitochondria, tiny powerhouses inside nearly every cell of the body. Depending on the affected genes and cell types, the diseases can cause muscle weakness, liver disease, diabetes, seizures, developmental delays or vision problems. Existing therapies aim to ease the symptoms or slow the progression of the diseases, but can't entirely cure them.

In their new work, Belmonte and collaborators from around the world collected skin samples from patients with mitochondrial encephalomyopathy or Leigh Syndrome, both severe disorders that affect the brain and muscles.

The teams began by using current standard protocols to derive pluripotent stem cells from the skin cells, a process that resets the cells to their most basic state.

"During the process of stem cell generation, you spontaneously get different types of clones," says Alejandro Ocampo, a research associate in Izpisua Belmonte's lab and one of the authors of the new paper. If the patient cells have an initial mix of healthy and diseased mitochondria, healthy and diseased stem cells will be generated. Then, the stem cells with healthy mitochondria can be picked out.

For some patients, though, this straightforward approach doesn't work; their cells don't have enough--or any--healthy mitochondria to start with.

So the team came up with a second approach: move the nucleus of the patient's skin cells, which contains most of their genes, into a donor egg cell with healthy mitochondria. Then, use the new egg cell to generate pluripotent stem cells. When the researchers did this, they found the healthy mitochondria took over, and healthy, genetically similar cells from the patient were successfully generated.

"In either case, the idea is that we have healthy stem cells, and we know how to convert pluripotent stem cells into different cell types," says Jun Wu, an author of the paper and research associate in Izpisua Belmonte's lab. "They have the potential to give rise to every cell type in the body."

For now, that means that researchers can use the healthy cells to generate heart, brain, muscle or eye cells from the mutation-free stem cells. But methods to make those cells fully mature and functional and transplant them into patients are still under development.

The new method will also be a boon to basic research, Izpisua Belmonte adds. Scientists have long struggled to understand why different organs and tissues are affected so differently by mitochondrial mutations. By comparing stem cells with mitochondrial mutations with healthy ones, and coaxing each to develop into different cell types, they can study this aspect of mitochondrial diseases in more detail.

###

Other researchers on the study were Li Ma of the Salk Institute; Hong Ma, Riffat Ahmed, Eunju Kang, Yeonmi Lee, Tomonari Hayama, Ying Li Crystal Van Dyken, Nuria Marti Gutierrez, Rebecca Tippner-Hedges, Amy Koski, Nargiz Mitalipov, Paula Amato, Don P. Wolf, and Shoukhrat Mitalipov of Oregon Health & Science University; Clifford D. L. Folmes, and Andre Terzic of the Mayo Clinic; Robert Morey, Sergio Mora-Castilla, and Louise C. Laurent of the University of California, San Diego; Joanna Poulton of the University of Oxford; and Xinjian Wang and Taosheng Huang of Cincinnati Children's Hospital.

The work was supported by the G. Harold and Leila Y. Mathers Charitable Foundation and the Leona M. and Harry B. Helmsley Charitable Trust.

About the Salk Institute for Biological Studies:

The Salk Institute for Biological Studies is one of the world's preeminent basic research institutions, where internationally renowned faculty probes fundamental life science questions in a unique, collaborative and creative environment. Focused both on discovery and on mentoring future generations of researchers, Salk scientists make groundbreaking contributions to our understanding of cancer, aging, Alzheimer's, diabetes and infectious diseases by studying neuroscience, genetics, cell and plant biology, and related disciplines. Faculty achievements have been recognized with numerous honors, including Nobel Prizes and memberships in the National Academy of Sciences. Founded in 1960 by polio vaccine pioneer Jonas Salk, MD, the Institute is an independent nonprofit organization and architectural landmark.

Media Contact

Salk Communications
press@salk.edu
858-453-4100

 @salkinstitute

http://www.salk.edu 

Salk Communications | EurekAlert!

More articles from Life Sciences:

nachricht How brains surrender to sleep
23.06.2017 | IMP - Forschungsinstitut für Molekulare Pathologie GmbH

nachricht A new technique isolates neuronal activity during memory consolidation
22.06.2017 | Spanish National Research Council (CSIC)

All articles from Life Sciences >>>

The most recent press releases about innovation >>>

Die letzten 5 Focus-News des innovations-reports im Überblick:

Im Focus: Can we see monkeys from space? Emerging technologies to map biodiversity

An international team of scientists has proposed a new multi-disciplinary approach in which an array of new technologies will allow us to map biodiversity and the risks that wildlife is facing at the scale of whole landscapes. The findings are published in Nature Ecology and Evolution. This international research is led by the Kunming Institute of Zoology from China, University of East Anglia, University of Leicester and the Leibniz Institute for Zoo and Wildlife Research.

Using a combination of satellite and ground data, the team proposes that it is now possible to map biodiversity with an accuracy that has not been previously...

Im Focus: Climate satellite: Tracking methane with robust laser technology

Heatwaves in the Arctic, longer periods of vegetation in Europe, severe floods in West Africa – starting in 2021, scientists want to explore the emissions of the greenhouse gas methane with the German-French satellite MERLIN. This is made possible by a new robust laser system of the Fraunhofer Institute for Laser Technology ILT in Aachen, which achieves unprecedented measurement accuracy.

Methane is primarily the result of the decomposition of organic matter. The gas has a 25 times greater warming potential than carbon dioxide, but is not as...

Im Focus: How protons move through a fuel cell

Hydrogen is regarded as the energy source of the future: It is produced with solar power and can be used to generate heat and electricity in fuel cells. Empa researchers have now succeeded in decoding the movement of hydrogen ions in crystals – a key step towards more efficient energy conversion in the hydrogen industry of tomorrow.

As charge carriers, electrons and ions play the leading role in electrochemical energy storage devices and converters such as batteries and fuel cells. Proton...

Im Focus: A unique data centre for cosmological simulations

Scientists from the Excellence Cluster Universe at the Ludwig-Maximilians-Universität Munich have establised "Cosmowebportal", a unique data centre for cosmological simulations located at the Leibniz Supercomputing Centre (LRZ) of the Bavarian Academy of Sciences. The complete results of a series of large hydrodynamical cosmological simulations are available, with data volumes typically exceeding several hundred terabytes. Scientists worldwide can interactively explore these complex simulations via a web interface and directly access the results.

With current telescopes, scientists can observe our Universe’s galaxies and galaxy clusters and their distribution along an invisible cosmic web. From the...

Im Focus: Scientists develop molecular thermometer for contactless measurement using infrared light

Temperature measurements possible even on the smallest scale / Molecular ruby for use in material sciences, biology, and medicine

Chemists at Johannes Gutenberg University Mainz (JGU) in cooperation with researchers of the German Federal Institute for Materials Research and Testing (BAM)...

All Focus news of the innovation-report >>>

Anzeige

Anzeige

Event News

Plants are networkers

19.06.2017 | Event News

Digital Survival Training for Executives

13.06.2017 | Event News

Global Learning Council Summit 2017

13.06.2017 | Event News

 
Latest News

Quantum thermometer or optical refrigerator?

23.06.2017 | Physics and Astronomy

A 100-year-old physics problem has been solved at EPFL

23.06.2017 | Physics and Astronomy

Equipping form with function

23.06.2017 | Information Technology

VideoLinks
B2B-VideoLinks
More VideoLinks >>>