Thursday, June 28, 2012

Successful Transplant of Patient-Derived Stem Cells Into Mice With Muscular Dystrophy

Source: University College London
Date: 28 June 2012

Summary:

Stem cells from patients with a rare form of muscular dystrophy have been successfully transplanted into mice affected by the same form of dystrophy, according to a new study published today in Science Translational Medicine.

For the first time, scientists have turned muscular dystrophy patients’ fibroblast cells (common cells found in connective tissue) into stem cells and then differentiated them into muscle precursor cells. The muscle cells were then genetically modified and transplanted into mice. The new technique could be used in the future for treating patients with limb-girdle muscular dystrophy (a rare form in which the shoulders and hips are primarily affected) and, possibly, other forms of muscular dystrophies.

In this study, scientists focused on genetically modifying a type of cell called a mesoangioblast, which is derived from blood vessels and has been shown in previous studies to have potential in treating muscular dystrophy. However, the authors found that they could not get a sufficient number of mesoangioblasts from patients with limb-girdle muscular dystrophy because the muscles of the patients were depleted of these cells.

Instead, scientists in this study “reprogrammed” adult cells from patients with limb-girdle muscular dystrophy into stem cells and were able to induce them to differentiate into mesoangioblast-like cells. After these ‘progenitor’ cells were genetically corrected using a viral vector, they were injected into mice with muscular dystrophy, where they homed-in on damaged muscle fibres.

The researchers also showed that when the same muscle progenitor cells were derived from mice the transplanted cells strengthened damaged muscle and enabled the dystrophic mice to run for longer on a treadmill than dystrophic mice that did not receive the cells.

Human Model of Huntington's Disease Created from Skin's Stem Cells

Source: University of California - Irvine
Date: June 28, 2012

Summary:

An international consortium of Huntington's disease experts, including several from the Sue & Bill Gross Stem Cell Research Center at UC Irvine, has generated a human model of the deadly inherited disorder directly from the skin cells of affected patients. The re-created neurons, which live in a petri dish, will help researchers better understand what disables and kills brain cells in people with HD and let them gauge the effects of potential drug therapies on cells that are otherwise locked deep in the brain. The research is published online June 28 in the journal Cell Stem Cell.

Turning Skin Cells Into Brain Cells: Huntington's Disease in a Dish

Source: Johns Hopkins Medical Institutions
Date: June 28, 2012

Summary:

Johns Hopkins researchers, working with an international consortium, say they have generated stem cells from skin cells from a person with a severe, early-onset form of Huntington's disease (HD), and turned them into neurons that degenerate just like those affected by the fatal inherited disorder.

To conduct their experiment, researchers took a skin biopsy from a patient with very early onset HD.When seen by Ross at the HD Center at Hopkins, the patient was just seven years old. She had a very severe form of the disease, which rarely appears in childhood, and of the mutation that causes it. Using cells from a patient with a more rapidly progressing form of the disease gave Ross' team the best tools with which to replicate HD in a way that is applicable to patients with all forms of HD.

Her skin cells were grown in culture and then reprogrammed in a lab into induced pluripotent stem cells. Scientists converted those cells into generic neurons and then into medium spiny neurons. What they found was that the medium spiny neurons deriving from HD cells behaved just as they expected medium spiny neurons from an HD patient would. They showed rapid degeneration when cultured in the lab using basic culture medium without extensive supporting nutrients. By contrast, control cell lines did not show neuronal degeneration.

The research, published in the journal Cell Stem Cell, is the work of a Huntington's Disease iPSC Consortium, including scientists from the Johns Hopkins University School of Medicine in Baltimore, Cedars-Sinai Medical Center in Los Angeles and the University of California, Irvine, as well as six other groups.

Wednesday, June 27, 2012

Regulation of Telomerase in Stem Cells and Cancer Cells

Source: Max-Planck-Gesellschaft
Date: June 27, 2012

Summary:

Scientists at the Max Planck Institute of Immunobiology and Epigenetics have gained important insights for stem cell research which are also applicable to human tumours and could lead to the development of new treatments. Researchers discovered a molecular link exists between the telomerase that determines the length of the telomeres and a signalling pathway known as the Wnt/β-signalling pathway.

The researchers demonstrated that β-catenin regulates the telomerase gene directly, and has explained the molecular mechanism at work here. Embryonic stem cells with mutated β-catenin generate more telomerase and have extended telomeres, while cells without β-catenin have low levels of telomerase and have shortened telomeres. This regulation mechanism can also be found in human cancer cells. These discoveries could lead to the development of a new approach to the treatment of human tumours.

New Approach to Reverse Multiple Sclerosis in Mice Models

Source: Mayo Clinic
Date: June 27, 2012

Summary:

Mayo Clinic researchers have successfully used smaller, folded DNA molecules to stimulate regeneration and repair of nerve coatings in mice that mimic multiple sclerosis (MS). They say the finding, published June 28 in the journal PLoS ONE, suggests new possible therapies for MS patients.

Stem Cells Can Beat Back Diabetes

Source: University of British Columbia
Date: June 27, 2012

Summary:

University of British Columbia scientists, in collaboration with an industry partner, have successfully reversed diabetes in mice using stem cells, paving the way for a breakthrough treatment for a disease that affects nearly one in four Canadians.

The research is the first to show that human stem cell transplants can successfully restore insulin production and reverse diabetes in mice. Crucially, they re-created the “feedback loop” that enables insulin levels to automatically rise or fall based on blood glucose levels. The study is published online today in the journal Diabetes.

After the stem cell transplant, the diabetic mice were weaned off insulin, a procedure designed to mimic human clinical conditions. Three to four months later, the mice were able to maintain healthy blood sugar levels even when being fed large quantities of sugar. Transplanted cells removed from the mice after several months had all the markings of normal insulin-producing pancreatic cells.

Sunday, June 24, 2012

Blood-Brain Barrier Building Blocks Forged from Human Stem Cells

Source: University of Wisconsin-Madison
Date: June 24, 2012

Summary:

The blood-brain barrier -- the filter that governs what can and cannot come into contact with the mammalian brain -- is a marvel of nature. It effectively separates circulating blood from the fluid that bathes the brain, and it keeps out bacteria, viruses and other agents that could damage it. But the barrier can be disrupted by disease, stroke and multiple sclerosis, for example, and also is a big challenge for medicine, as it can be difficult or impossible to get therapeutic molecules through the barrier to treat neurological disorders.

Now, however, the blood-brain barrier may be poised to give up some of its secrets as researchers at the University of Wisconsin-Madison have created in the laboratory dish the cells that make up the brain's protective barrier. Writing in the June 24, 2012 edition of the journal Nature Biotechnology, the Wisconsin researchers describe transforming stem cells into endothelial cells with blood-brain barrier qualities.

The research team coaxed both embryonic and induced pluripotent stem cells to form the endothelial cells of the blood-brain barrier. The use of induced cells, which can come from patients with specific neurological conditions, may be especially important for modeling disorders that compromise the blood-brain barrier. What's more, because the cells can be mass produced, they could be used to devise high-throughput screens for molecules that may have therapeutic value for neurological conditions or to identify existing drugs that may have neurotoxic qualities.

Friday, June 22, 2012

Speeding Up Bone Growth by Manipulating Stem Cells

Source: University of South Carolina
Date: June 22, 2012

Summary:

A researcher at the University of South Carolina, has made significant progress toward reducing the time for broken bone to heal. A study published in Molecular Pharmaceutics found that surfaces coated with bionanoparticles could greatly accelerate the early phases of bone growth. Their coatings, based in part on genetically modified Tobacco mosaic virus, reduced the amount of time it took to convert stem cells into bone nodules -- from two weeks to just two days.

The conversion of these cells -- called stem cells -- is set into motion by external cues. In bone healing, the body senses the break at the cellular level and begins converting stem cells into new bone cells at the location of the break, bonding the fracture back into a single unit. The process is very slow, which is helpful in allowing a fracture to be properly set, but after that point the wait is at least an inconvenience, and in some cases highly detrimental. The researchers found that the coatings alone could reduce the amount of time to grow bone nodules from stem cells. Since then, theyhave refined their approach to better define just what it is that accelerates bone growth.

Wednesday, June 20, 2012

Discovery of ‘Master Molecule’ Could Improve Stem Cell Treatment for Heart Attacks

Source: Johns Hopkins University
Date: June 20, 2012

Summary:

Johns Hopkins researchers have discovered that a single protein molecule may hold the key to turning cardiac stem cells into blood vessels or muscle tissue, a finding that may lead to better ways to treat heart attack patients.

Human heart tissue does not heal well after a heart attack, instead forming debilitating scars. However, for reasons not completely understood, stem cells can assist in this repair process by turning into the cells that make up healthy heart tissue, including heart muscle and blood vessels. Recently, doctors elsewhere have reported promising early results in the use of cardiac stem cells to curb the formation of unhealthy scar tissue after a heart attack. But the discovery of a “master molecule” that guides the destiny of these stem cells could result in even more effective treatments for heart patients, the Johns Hopkins researchers say.

In a study published in the June 5 online edition of journal Science Signaling, the team reported that tinkering with a protein molecule called p190RhoGAP shaped the development of cardiac stem cells, prodding them to become the building blocks for either blood vessels or heart muscle. The team members said that by altering levels of this protein, they were able to affect the future of these stem cells.

Tuesday, June 19, 2012

Understanding of Spinal Muscular Atrophy Improved With Use of Stem Cells

Source: Cedars-Sinai Medical Center
Date: June 19, 2012

Summary:

LOS ANGELES – Cedars-Sinai’s Regenerative Medicine Institute has pioneered research on how motor-neuron cell-death occurs in patients with spinal muscular atrophy, offering an important clue in identifying potential medicines to treat this leading genetic cause of death in infants and toddlers. The study, published in the June 19 online issue of PLoS ONE, extends the institute’s work to employ pluripotent stem cells to find a pharmaceutical treatment for spinal muscular atrophy or SMA, a genetic neuromuscular disease characterized by muscle atrophy and weakness.

New Method Generates Cardiac Muscle Patches from Stem Cells

Source: University of Michigan Health System
Date: June 19, 2012

Summary:

A cutting-edge method developed at the University of Michigan Center for Arrhythmia Research successfully uses stem cells to create heart cells capable of mimicking the heart's crucial squeezing action. The cells displayed activity similar to most people's resting heart rate. At 60 beats per minute, the rhythmic electrical impulse transmission of the engineered cells in the U-M study is 10 times faster than in most other reported stem cell studies.

An image of the electrically stimulated cardiac cells is displayed on the cover of the current issue of Circulation Research, a publication of the American Heart Association. For those suffering from common, but deadly heart diseases, stem cell biology represents a new medical frontier. The U-M team of researchers is using stem cells in hopes of helping the 2.5 million people with an arrhythmia, an irregularity in the heart's electrical impulses that can impair the heart's ability to pump blood.

Their objective included developing a bioengineering approach, using stem cells generated from skin biopsies, which can be used to create large numbers of cardiac muscle cells that can transmit uniform electrical impulses and function as a unit. Furthermore, the team designed a fluorescent imaging platform using light emitting diode (LED) illumination to measure the electrical activity of the cells.

Wednesday, June 13, 2012

'Magical State' of Embryonic Stem Cells May Help Overcome Hurdles to Therapeutics

Source: Salk Institute for Biological Studies
Date: June 13, 2012

Summary:

LA JOLLA, CA—With their potential to treat a wide range of diseases and uncover fundamental processes that lead to those diseases, embryonic stem (ES) cells hold great promise for biomedical science. A number of hurdles, both scientific and non-scientific, however, have precluded scientists from reaching the holy grail of using these special cells to treat heart disease, diabetes, Alzheimer's and other diseases.

In a paper published June 13 in Nature, scientists at the Salk Institute for Biological Studies report discovering that ES cells cycle in and out of a "magical state" in the early stages of embryo development, during which a battery of genes essential for cell potency (the ability of a generic cell to differentiate, or develop, into a cell with specialized functions) is activated. This unique condition, called totipotency, gives ES cells their unique ability to turn into any cell type in the body, thus making them attractive therapeutic targets.

Tuesday, June 12, 2012

A New Way to Make Bone: Fresh, Purified Fat Stem Cells Grow Bone Better, Faster

Source: University of California, Los Angeles (UCLA), Health Sciences
Date: June 12, 2012

Summary:

UCLA stem cell scientists who purified a subset of stem cells from fat tissue and used the stem cells to grow bone discovered that the bone formed faster and was of higher quality than bone grown using traditional methods. The finding may one day eliminate the need for painful bone grafts that use material taken from patients during invasive procedures. The study was published June 11 in the early online edition of Stem Cells Translational Medicine.

Thursday, June 07, 2012

Scientists Reprogram Skin Cells Into Brain Cells

Source: Gladstone Institutes
Date: June 7, 2012

Summary:

Scientists at the Gladstone Institutes have for the first time transformed skin cells -- with a single genetic factor -- into cells that develop on their own into an interconnected, functional network of brain cells. The research offers new hope in the fight against many neurological conditions because scientists expect that such a transformation -- or reprogramming -- of cells may lead to better models for testing drugs for devastating neurodegenerative conditions such as Alzheimer's disease.

This research comes at a time of renewed focus on Alzheimer's disease, which currently afflicts 5.4 million people in the United States alone -- a figure expected to nearly triple by 2050. Yet there are no approved medications to prevent or reverse the progression of this debilitating disease.

In findings appearing online June 7 in Cell Stem Cell, researchers describe how they transferred a single gene called Sox2 into both mouse and human skin cells. Within days the skin cells transformed into early-stage brain stem cells, also called induced neural stem cells (iNSCs). These iNSCs began to self-renew, soon maturing into neurons capable of transmitting electrical signals. Within a month, the neurons had developed into neural networks.

Scientists Reprogram Skin Cells into Brain Cells Innovative technique lays groundwork for novel stem cell therapies

Source: Gladstone Institutes
Date: June 7, 2012

Summary:

SAN FRANCISCO, CA—Scientists at the Gladstone Institutes have for the first time transformed skin cells—with a single genetic factor—into cells that develop on their own into an interconnected, functional network of brain cells. The research offers new hope in the fight against many neurological conditions because scientists expect that such a transformation—or reprogramming—of cells may lead to better models for testing drugs for devastating neurodegenerative conditions such as Alzheimer's disease.

In findings appearing online today in Cell Stem Cell, researchers in the laboratory of Gladstone Investigator Yadong Huang, MD, PhD, describe how they transferred a single gene called Sox2 into both mouse and human skin cells. Within days the skin cells transformed into early-stage brain stem cells, also called induced neural stem cells (iNSCs). These iNSCs began to self-renew, soon maturing into neurons capable of transmitting electrical signals. Within a month, the neurons had developed into neural networks.

Wednesday, June 06, 2012

The Real Culprit Behind Hardened Arteries? Stem Cells, Says Landmark Study

Source: University of California - Berkeley
Date: June 6, 2012

Summary:

BERKELEY — One of the top suspects behind killer vascular diseases is the victim of mistaken identity, according to researchers from the University of California, Berkeley, who used genetic tracing to help hunt down the real culprit. The UC Berkeley researchers say that these newly discovered stem cells contribute to artery-hardening vascular diseases that can lead to heart attacks and strokes.

The guilty party is not the smooth muscle cells within blood vessel walls, which for decades was thought to combine with cholesterol and fat that can clog arteries. Blocked vessels can eventually lead to heart attacks and strokes, which account for one in three deaths in the United States.

Instead, a previously unknown type of stem cell — a multipotent vascular stem cell — is to blame, and it should now be the focus in the search for new treatments, the scientists report in a new study appearing June 6 in the journal Nature Communications.

Tuesday, June 05, 2012

Neuralstem Updates ALS Stem Cell Trial Progress; Emory University Institutional Review Board Approves Amendment

Source: Neuralstem, Inc.
Date: June 5, 2012

Summary:

ROCKVILLE, Md., /PRNewswire/ -- Neuralstem, Inc. announced that the Emory University Institutional Review Board (IRB) approved the amendment to the ongoing Phase I trial evaluating Neuralstem's spinal cord stem cells in the treatment of amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease). The amendment permits the return of three previously-treated patients to the trial to receive additional injections of cells. This modification to the protocol was approved earlier by the Food and Drug Administration (FDA). Implementation was contingent upon IRB approval, which has now been secured.