In an exciting new development, the Food and Drug Administration (FDA) has approved a new stem cell-derived treatment for a phase I clinical trial in the treatment of multiple sclerosis (MS). This FDA approval is the culmination of more than a decades worth of research into the therapeutic potential of stem cells for MS patients by the stem cell research division of the Tisch Multiple Sclerosis Research Center of New York. The first of its kind in the United States, the study will initially enroll 20 progressive MS patients, recruited from the existing patient population of the International Multiple Sclerosis Management Practice, who will have specialized neural progenitor cells injected into their cerebrospinal fluid in order to directly target regenerative mechanisms in the central nervous system. Saud Sadiq, Director of TMSRC, collaborates with NYSCF's multiple sclerosis team, led by Dr. Valentina Fossati, by recruiting patient participants for Dr. Fossati’s work generating stem cells from MS patients.
In an large step forward for neuroscience research around the globe, scientists at Institute of Molecular Biotechnology at the Austrian Academy of Science in Vienna created mini-brain-like organoids using stem cells. Published in Nature yesterday, these pea-sized clusters of human brain tissue can help researchers explore important questions about brain development and neurological disorders. Related to this exciting development in the field, NYSCF scientists are generating neural models “in the dish” using stem cells to create and develop neurons that interact with each other and can be measured on a molecular level using sensitive electronic equipment. This type of research is critical for diseases such as Alzheimer’s and Parkinson’s, among many others.
Japan's regulatory body has given final go-ahead for the first-ever clinical trial testing patient-derived stem cells to treat age-related macular degeneration (AMD), a common form of blindness. Six patients will be enrolled initially by the Riken Center for Developmental Biology and the Institute of Biomedical Research and Innovation Hospital in Kobe. They will donate a small section of skin from which researchers will derive induced pluripotent stem (iPS) cells--a type of embryonic-like cell genetically matched to the patient. Thereafter, the scientists will then transform these cells into healthy eye cells to replace those damaged in AMD, and then transplant the grafts into patients. This pioneering trial will first test the safety and tolerability of the grafts.
In 2005, Dr. Shinya Yamanak reported that the addition of four genes could "turn back the clock" on adult cells to become embryonic-like stem cells, known as induced pluripotent stem (iPS) cells. Significantly, iPS cells are patient-specific, renew indefinitely, and can become any of the body's other cell types. This method, while opening up an entire new field of research, introduces new, potentially cancer-causing genes into the cells, limiting clinical application.
To overcome this caveat, scientists have been fine-tuning the reprogramming process to procure iPS cells without added genes. Reported in Science, a Chinese group discovered how the right chemical combination can change skin cells into pluripotent stem cells, or CiPS. As researchers further refine this chemicals-only approach, these CiPS could be applied to various regenerative medicine applications.
Stem cell-derived liver cells, cultured in a Petri dish alongside blood and connective tissue, spontaneously assembled into a human liver bud, closely resembling a five to six week old human liver. This advance, reported in Nature, represents the first time a vascularized, three-dimensional organ has been produced from induced pluripotent stem (iPS) cells. Transplanted into mice, the buds broke down drugs that human, not mouse, livers can process.
Crossing from science fiction to science, new findings in Nature pave the way for limb regeneration. Researchers led by Mayumi Ito of New York University have isolated active stem cells in the base of mouse nail beds, which spring into action when digits are clipped. Importantly, the activity of these stem cells is regulated by the same molecular pathways as in reptiles that can regenerate entire limbs. However, if nails are completely clipped or there is little remaining tissue immediately below the nail, re-growth fails in mice.
Learning new songs tipped researchers off to the unique ability of canaries to add new brain cells. Now, based on these findings, researchers have learned how to trigger brain cell growth in mice with Huntington’s disease. In patients with this neurodegenerative disease, brain cells die off, leading to a full range of movement, mood, and behavioral symptoms. The generation of new brain cells could, in theory, stymie the progression of this disease. Dr. Steve Goldman of the University of Rochester Medical Center and his team hope to translate their findings, reported in Cell Stem Cell, to patients.
Through stem cell techniques, a group of scientists led by Dr. Anita Bhattacharyya generated neurons from Down's syndrome patients' skin samples, providing an unprecedented model of early brain development in Down's syndrome. The neurons formed fewer connections early-on than those without the disorder. This difference, according to the researchers, is critical and could help explain features of Down's syndrome. When examining genes, they found certain age-related genes to be more active in the Down's syndrome neurons than those of unaffected participants. Taken together, these findings using Down's syndrome cell models provide initial key findings that may yield therapies.
Fueling hopes of patients with heart damage, a 2001 Nature study showed regrowth of heart tissue in mice. Subsequently refuted, the results pointed researchers to different stem cell-based strategies to treat heart damage. The BBC provides an overview of these approaches--ranging from injecting stem cells to directly converting cells into heart muscle.
Scientists from UCLA have succeeded in modelling ataxia telangiectasia (A-T), a rare genetic disorder, in a dish. This neurodegenerative disease affects 1 in 100,000 worldwide, with symptoms that range from difficulty in controlling movement to delayed development. A-T patients are at increased risk for cancer, lung disease, and diabetes. Mouse models, while providing insight into A-T, fail to fully reflect all aspects of this disease. In this study, the scientists coaxed A-T patients' skin cells to become induced pluripotent stem (iPS) cells, which became the type of neurons affected by this disease. The team aims to use these cells to better understand the mechanisms of this disease and to test drug-like chemical compounds.
Scientists report the successful creation of patient-specific human embryonic stem cells with just an egg cell and a skin sample through a technique called somatic cell nuclear transfer (SCNT). NYSCF celebrates this major advance for the field that we anticipate will lead to advancements in uncovering new disease mechanisms and to personalized treatments and cures for disease.
Researchers, including a group at NYSCF, have conducted research to transfer the gene-containing nucleus of an adult cell into an egg cell to generate an early-stage embryo, called a blastocyst, and then derive stem cell lines. In 2011, NYSCF scientists paved the way for this discovery and achieved the proof-of-concept: Dieter Egli, PhD, and his team derived the first-ever stem cell lines through this method, yet the cells were triploid, containing an extra set of chromosomes.
To generate stem cell lines with only two sets of chromosomes, Shoukhrat Mitalipov, PhD, and his team at the Oregon Health and Science University first optimized the SCNT protocol in nonhuman primate cells. Selecting previously successful techniques, the scientists promoted blastocyst development by exposing the egg cells to both an electrical pulse and caffeine. They procured several lines of nuclear transfer embryonic stem cells (NT-ESCs).
Then translated to human cells, the group extended their approach across several egg donors with skin cells taken from a patient with Leigh syndrome (a lethal mitochondrial disease). The resultant NT-ESCs were fully pluripotent, meaning that they could become any of the other cell types that compose the body, contained the correct chromosome count, and lacked mitochondrial DNA of the skin cells.
NT-ESCs confer several advantages over other pluripotent stem cell sources. They, unlike embryonic stem cells, are patient-specific, and they carry fewer, potentially dangerous genetic changes than induced pluripotent stem cells, or cells derived from other adult cell types like skin. Importantly for cell therapies, NT-ESCs can be employed irrespective of a donor’s mitochondrial DNA.
NYSCF, one of the only research institutes in the country involved in this line of research, looks forward to the potential this technique holds develop cures and treatments for patients suffering from diseases.
A two and a half year old girl has become the youngest recipient of a bioengineered wind pipe, made from her own stem cells. Born without a trachea, she had relied on an tube to breathe. Clinicians and scientists engineered a biodegradable plastic implant, which was then seeded with her own bone marrow stem cells. Once tissue matured in a bioreactor, surgeons transplanted the trachea, enabling her to breathe on her own for the first time. While the procedure does not have FDA approval, the scientists remain optimistic that further successes could lead to widespread treatment.
Led by Douglas Melton, PhD, Harvard Stem Cell Institute Principal Faculty member and NYSCF Medical Advisory Board Executive Committee member, a new Cell study identifies a hormone in mice that contributes to the rapid proliferation of insulin-producing beta cells. In diabetes, beta cells malfunction and/or die, leading to a decreased ability to process sugars. While the disease can be managed by insulin injections and changes in diet and lifestyle, patients are at risk for related complications. Betatrophin, also found in humans, could be a potential target for type 2 diabetes treatments.
University of Oxford researchers report in Science self-assembling, tissue-like networks of water droplets. Fats enclose the water droplets akin to a cell’s own lipid bilayer. Deposited by a three-dimensional printer, this material may act as a biological scaffold to help grow or even replace human tissue.
A key challenge facing tissue engineers has been providing tissue with blood flow. Now, a group of scientists at the University of Michigan have identified the source of previous failed attempts to encourage tissue vascularization with cells. The quality of injected cells appears to be tied to how well blood vessels branch off and grow. Adult stem cells with scaffolding material form more robust vessels than conventional cells. These findings appear in Tissue Engineering Part A.
“New blood” takes on a literal meaning as researchers report the rejuvenation of mouse blood cells in Blood. The Lund University scientists reprogrammed stem cells that produce blood, effectively cancelling-out age-related changes to these stem cells. Old blood-forming stem cells become less able to mature into blood and immune cells.
They’re not just support cells, astrocytes play an important role in memory and learning according to a new Cell Stem Cell study. Steven Goldman and colleagues derived neural progenitor cells from human induced pluripotent stem (iPS) cells and then transplanted these cells into the brains of newborn mice. While most of the cells remained immature, some developed into astrocytes. These brain-cell endowed mice demonstrated superior maze-solving skills and recognized objects in new locations compared to non-chimeric mice. This study could help clue in neuroscientists to the evolution of astrocytes and their role in the human brain.
Moving toward personalized therapies, researchers report the successful, matched transplantation of autologous rhesus monkey induced pluripotent stem (iPS) cells back into the brain. These genetically engineered monkeys suffer from balance disturbances and display other symptoms similar to human Parkinson’s patients. Led by Su-Chun Zhang at the University of Wisconsin, the team derived neural progenitors from parkinsonian monkeys’ skin samples, and then allowed the cells to fully mature into the brain. Tagged with fluourescent markers, the transplanted cells differentiated into a spectrum of brain cells—astrocytes, dopaminergic neurons, among others. On a promising note, the transplanted cells elicited a minimal immune response and the monkeys showed no signs of cancer at six month follow-up according to this Cell Reports study.
As stem cell research gets closer to the clinic, the delivery of cell therapies lags behind. To transplant stem cell-derived treatments a flexible and precise tool instrument is necessary. Neurosurgeon and stem cell scientist Daniel Kim have developed a bendable needle that can successfully inject cells, even electrodes, into the brains of mice. While human brains are more complex, the needle is compatible with MRIs and uses an advanced computer system to accurately deposit materials to the wanted site.
Combining advances in gene therapy and stem cell technology, researchers report a potential treatment for a mouse model of Duchenne muscular dystrophy (DMD), a fatal genetic disease. Rita Perlgeiro with Michael Kyba and colleagues engineered mice with a mutation to the dystrophin and utrophin genes, much like human DMD counterparts. They then generated induced pluripotent stem (iPS) cell lines from mouse skin cells and employed a special gene-correcting tool called Sleeping Beauty Transposon to deliver the correct copy of the utrophin gene. Once integrated into the skin cells’ DNA, the researchers differentiated these cells into muscle cells for transplantation back into the DMD mice. Exceeding expectations, the iPS-derived muscle cells engrafted into disease sites and helped reduce symptoms of DMD.
Induced pluripotent stem (iPS) cells are slated to enter a clinical study in Japan for the treatment of age-related macular degeneration. Massayo Takahashi, an opthamologist at RIKEN Center for Developmental Biology and leader of this project, plans to treat six patients who suffer from this degenerative eye disease that commonly leads to blindness. Takahashi and her team will derive iPS cells from the participants’ skin samples and generate replacement retinal cells for transplantation into the retina. While safety concerns linger and some scientists harbor reservations on efficacy, Japan’s regulatory scheme will likely enable the study to move forward. If this iPS cell therapy proves safe and effective, it could enter formal clinical trials and treat patients in the clinic soon.
For the first time, researchers led by Hans Clevers and Markus Grompe have successfully identified liver stem cells from a mouse. Reported in Nature, the team worked off a hypothesis that a previously identified marker of small intestine and colon stem cells could also mark liver stem cells. They could thereby isolate these liver stem cells and expand them in culture. Upon transplantation of these cells into mice with cirrhosis, there was a moderate therapeutic benefit.
Every dollar invested in the Human Genome Project returned $140 to the US economy. Now, President Obama will unveil a similar, ambitious plan to map the human brain in March. A multi-institutional, cross-field effort will build on basic research to better understand the brain and related disease pathologies. The ultimate goal of the Human Brain Activity Map is to find better treatments and cures to degenerative neurological disease.
Subtle yet important, the interactions between motor neurons with neighboring cell types contribute to the pathologies of ALS (or Lou Gehrig’s disease), specifically motor neuron death. Reported in Proceedings of the National Academy of Sciences, mutations to a gene called TDP-43, although rare, cause astrocyte cell death. While astrocyte cell death is associated with motor neuron death, they are not directly toxic. Siddharthan Chardan, who led the study, generated induced pluripotent stem cells from ALS patients to make this discovery.
It just takes the repression of one protein to directly transdifferentiate skin cells into functional neurons according to a new study in Cell. Xiang-Dong Fu with a team of UCSD and Wuhan University scientists demonstrated that micro-RNAs silence a protein, which is sufficient to convert adult skin cells into neuron-like cells. The researchers are hopeful that this technique could lead to new drugs to treat degenerative diseases.
Make no bones about it: engineering a material to repair bone tissue is complex. A newly developed polymer that can be seeded with stem cells may help support damaged bone. Mark Bradley and colleagues describe this osteogenic material in Advanced Functional Materials. Next steps include demonstrating the feasibility of this polymer for the repair of bone damage.
Multiple sclerosis, an autoimmune disease in which immune cells attack the fatty protective layer of myelin cells, may have a potential treatment. Through stem cell techniques, a team led by Steven Goldman report in Cell Stem Cell the generation of myelin cells from human skin cells. The researchers implanted these new myelin cells in the brains of young, myelin-deficient mice, which greatly increased mouse survival. However, the successful hypothetical transplantation of these cells in human multiple sclerosis patients does not eliminate the immune system’s siege on this cell type. An application of this therapy would likely have to complement other treatments or performed repeatedly.
Taste receptor cells have a quick turnover rate. Every 10-16 days, new cells replace the old; yet, pinpointing the replenishing stem cell source has eluded researchers. In a study published in Stem Cells, researchers found a marker that identifies these cells. Significantly, these stem cells could be re-activated following taste receptor loss in cancer patients or the elderly. Peihua Jiang led the investigation.
Brown fat, commonly thought as long-term energy source for hibernating animals, also plays an important role in blood sugar regulation and day-to-day metabolism. Previous studies have demonstrated a positive association between brown fat and leanness; thereby, adding to a patient’s brown fat reserves could potentially treat obesity. A study in Cell Metabolism shows how skeletal muscle stem cells could develop into brown fat cells. In mice, the injection of a drug that reduces miRNA-133 levels led to increased brown fat and leanness. Michael Rudnicki and colleagues are optimistic that this treatment could become a viable therapy to fight against the current obesity epidemic.
A new 3D printer technology deposits human embryonic stem cells in droplets. Building on 3D printer technology, this new platform can eject these fragile cells, which could help one day grow complex tissues. A Biofabrication study details an initial investigation of this printer’s capabilities. .
Radiation exposure often triggers cell death in hematopoetic, or blood-forming, stem cells. A study in Nature Medicine shows that the addition of a hormone following exposure could help prevent the loss of these essential progenitors. Co-authors David Kirsch and John Chute launched their investigation with genetically engineered mice that lacked genes to regulate death of endothelial (or blood vessel lining) cells. When exposed to radiation, these mice fared better than their wild type counterparts, and, notably, these former mice expressed high levels of epidermal growth factor (EGF). The team then fortified bone marrow transplants with EGF. Those mice that received the solution survived at a high rate following a lethal radiation exposure. Potentially, this research could be translated to humans as a therapy following accidental radiation exposure or radiotherapy for cancer.
A team led by Heather Young and John Furness has demonstrated a possible cell therapy to treat gastrointestinal motility disorders like Hirschsprung disease. For patients with these diseases, the nerves that signal the digestive system to contract are not present, and surgery is required to correct this deficit. As reported in The Journal of Clinical Investigation, the transplantation of neural progenitor from mouse embryos can migrate and proliferate to the nerve-less gut in a mouse model.
Under the cover of a mesenchymal stem cell, the tuberculosis (TB) bacterium persists, even years after living asymptomatically. A new study in Science Translational Medicine provides evidence for this “wolf-in-stem-cell-clothing” model. Researchers led by Dean Felsher and Antonio Campos-Neto worked off the hunch of Bikul Das, first author on the study. Das spent years as a clinician in India, where he observed TB bacteria in bone marrow samples of patients. The researchers initially studied a mouse model of disease, finding that the TB bacterium hid in a stem cell niche, and expanded the investigation to patients in India, who showed signs of the bacteria in stem cells obtained from the bone marrow.
Arrhythmogenic right ventricular dysplasia/cadiomyopathy is a rare, inherited, and currently untreatable condition, which is a common cause of sudden death in adolescents. Researchers led by Daniel Judge and Huei-Sheng Vincent Chen generated cardiomyocytes from patients suffering from this condition; however, at first, they were unable to observe the cellular-level features of the disease in these immature cells. They developed, as reported in Nature, a method to trigger signs of the adult disease by activating the cells’ metabolism, producing a “disease in a dish.”
Approximately 20% of patients diagnosed with dry age-related macular degeneration develop the wet form, which leads to irreversible eye damage and eventual blindness. While the dry form can be diagnosed during an eye exam, there is currently no test that identifies the conversion from dry to wet. Per past studies, endothelial progenitor cells (EPCs) are elevated in the blood stream when new blood vessels form, a mechanism of disease for wet ARMD. Sai Chavala and colleagues developed a blood test to identify EPC levels in wet and dry ARMD patients with automated rare cell analysis (ARCA). Their results reported in PLOSOne show that the ARCA test might predict this transition to wet ARMD in dry ARMD patients.
Rats, immediately following an induced ischemic stroke, fared better when injected with allogenic mesenchymal stem cells than a placebo. While there was no evidence of stem cell proliferation at the infarct site, tests revealed increased cell proliferation and nearly normal behavioral function in the stem cell recipients, measured two weeks later. The study, led by Sebastian Cerdan and Exuperio Diez-Tejedor, was published in Stem Cell Research and Therapy.
Japanese researchers led by Kenji Osafune report the successful generation of mesoderm kidney tissue from induced pluripotent stem cells. This particular tissue type—between a stem and an adult kidney cell—was used to grow part of a urinary tubule. While safety issues need to be addressed, this study in Nature Communications could lead to new therapies for kidney disease.
Often, leukemia lays dormant before re-emerging. Researchers believe that a resistive subpopulation of leukemia stem cells underlie our current difficulty in eradicating this blood cancer. A new study in Cell Stem Cell reveals that, uniquely, leukemia stem cells have a “slower” metabolism than other tumor cells. Researchers led by Craig T. Jordan, applied a drug to target these cells’ metabolic pathway, effectively disrupting their ability to harness energy. These results may provide a new path to creating more effective leukemia treatments.
It’s not just humans who can reprogram cells. The leprosy bacterium can infect and thereafter revert adult Schwann cells into stem-like cells. The infection is spread by differentiation of the stem-like cells into skeletal muscle cells and by infected macrophages that consume these reprogrammed cells. Anura Rambukkana led the study, with results reported in Cell.
Children born with spinal muscular atrophy (SMA) have a grim prognosis: most infants rarely see it to their second birthday. This debilitating, genetic disease has no cure; however, a study in Science and Translational Medicine demonstrates a possible stem cell therapy. Giacomo Comi and colleagues derived stem cell lines from skin samples of patients with SMA, and corrected a faulty genetic copy of the gene responsible for the disease. The researchers then reprogrammed these corrected cells into motor neurons. Transplanted in mice, the motor neurons improved disease phenotype and increased lifespan.
Reported in Science and Translational Medicine, a light-activated hydrogel helps to repair damaged cartilage by acting as a scaffold for stem cells. Jennifer Elisseeff and collaborators modeled the activity of the gel in vitro, demonstrating cartilage tissue development. Following successful application of the hydrogel in a caprine (a goat-antelope) model, the group launched a pilot study to treat patients with cartilage damage to the medial femoral condyle, the lower part of the femur. Patients reported less pain, and imaging studies revealed healthy cartilage.
Duchenne muscular dystrophy (DMD), a rare genetic disorder, leads to muscle death and the formation of scar tissue due to a mutant copy of the dystrophin gene. Average DMD life expectancy is 25. While no cure exists, recent stem cell work in a mouse model reveals a possible therapy. Researchers led by Suzanne Berry-Miller generated aorta-derived mesoangioblast stem cells and then injected the cells into the hearts of dystrophin-deficient mice. The results, reported in Science and Translational Medicine, showed treatment delayed or prevented cardiac muscle damage.
Project REBORNE will commence clinical trials to help repair fractured bone in France. Following successful pre-clinical work and approval by the French Medicinal Agency, autologous mesenchymal stem cells in combination with a biomaterial will be transplanted into the fracture sites of 30 patients. Ultimately, the researchers hope to expand the study to other European centers to demonstrate the safety and tolerability of this intervention.
Hearing loss in mammals is thought irreversible: damage wrought to ear hair cells can lead to permanent deafness. A new study in Neuron from Albert Edge and colleagues uncovers a potential drug that regenerates ear hair cells. Previous research demonstrated that this compound could generate ear hair cells from stem cells, informing the decision of these researchers to apply the drug to the cochlea of deaf mice. Imaging and other analysis showed hearing recovery and the growth of new ear hair cells. This may be a promising therapeutic pathway to help deaf individuals.
The results of a Nature study may quell long-held concerns over the safety of transplanted induced pluripotent stem (iPS) cells. In a 2011 investigation, transplanted autologous iPS cells instigated an immune response in mice, a roadblock for the development of iPS cell therapies. However, Masumi Abe and colleagues demonstrated no difference in immune response between mice injected with undifferentiated iPS cells or embryonic stem (ES) cells or, significantly, skin or bone-marrow cells derived from iPS or ES cells. Nevertheless, the study may be limited by its methods in generating the differentiated bone-marrow cells from chimeric embryos, a technique that would not apply to a clinical setting.
A meta-analysis of 11 studies reveals that undifferentiated, mulitpotent neural stem cells (NSC) from mice or humans may slow the onset and mitigate the symptoms of ALS in mice. From these studies, Evan Snyder and fellow researchers concluded that the efficacy of this intervention was mediated by a range of factors. Of note, the transplanted cells benefited native neurons to produce their own protective molecules.
The Supreme Court refused to hear an appeal from the plaintiff’s of Sherley v. Sebelius. Significantly, this marks the end of a years-long legal battle to challenge the NIH’s ability to create guidelines for federally funded human embryonic stem cell research.
University of Oxford researchers report strides to reverse blindness in the Proceedings of the National Academy of Sciences. The group, led by Robert MacLaren, transplanted light-sensitive progenitor cells to mice with zero visual function, representing late-stage retinitis pigmentosa. Examination revealed formation of a light-sensitive photoreceptive layer as well as neural integration.
BRCA1, a gene commonly known for its role in cancer, has been found to play an essential role in hair follicle cell maintenance. As reported in Genes and Development, Cedic Blanpain and colleagues showed that deletion of this genes leads to eventual hair follicle stem cell death. Other cell types in the epidermis continued to proliferate despite this gene's deletion.
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A paradox that finally explains why certain breast cancers respond to treatment has been uncovered by Jian Jian Li and colleagues. For HER-2 negative breast cancers, often treatments crafted for HER-2 positive patients prove effective. A new study in Clinical Cancer Research reports that a radiotherapy-resistant group of HER-2 positive cancer stem cells are present in HER-2 negative breast cancers. These findings may help to elucidate treatments.
The expression of a single gene can directly convert heart muscle cells to cardiac pacemaker cells (i.e. SAN cells) both in an animal and a dish. An advantage over stem cell-derived pacemaker cells, the direct reprogramming method as outlined in a new Nature Biotechnology study eliminates risk of tumor growth. The Cedars-Sinai Heart Institute scientists led by Hee Cheol Cho, PhD, hope, in going forward with this research, that these cells could replace electronic pacemakers for patients with cardiovascular damage or disease.
To treat corneal blindness, University of Sheffield researchers led by Frederik Claeyssens have engineered a biodegradable material with pockets to hold stem cells in place as they help repair damage. The material mirrors the natural environment of corneal cells, an advantage over current corneal transplants. Details on the novel material are published in Acta Biomaterialia.
Pharmaceutical companies and academic institutions, with European Union backing, are collaborating to produce a bank of 1,500 induced pluripotent stem cell lines. This StemBANCC project, to be managed by the University of Oxford, will collect skin or blood samples from 500 individuals and derive stem cells to make available for research and drug development.
In Nature Methods, a group of researchers led by Duanqing Pei reported the derivation of neural progenitor cells from urine. Easily procured, human excreta could be a viable source of patient-specific stem cell lines. The researchers generated neurons and grafted the cells into the brains of newborn mice, which implanted and did not form tumors.
Induced pluripotent stem (iPS) cells derived from banked or even frozen blood samples present a practical and efficient platform to generate patient-specific cells. Researchers led by Professor Nicholas W. Morrell published in Stem Cells Translational Medicine, a proof-of-concept study. They isolated late-outgrowth endothelial progenitor cells from blood samples of both well donors and patients with hypertension, and then reprogrammed these cells into iPS cells.
Jasper, a dachshund, might not go on long walks, but he can stay in step on a treadmill with harness support after participation in a stem cell-based trial conducted by Wellcome Trust-MRC Stem Cell Institute researchers. Professor Robin Franklin and his team isolated olfactory ensheathing cells from the nasal cavity, which promote neuron replacement. These cells, after culture and expansion, were transplanted into the spinal chord injury site of half the study’s canine participants while the other half received a placebo injection. The cell dose group showed improved fore-hind coordination compared to the control dogs. Based off of this study, published in Brain, the researchers hope to have sufficient “proof of concept” to pursue similar studies.
To better understand genetic factors involved in brain development, researchers led by Tristan Bouschet and Pierre Vanderhaeghen examined mouse embryonic stem cells. When oncogene BCL6 was overexpressed, the stem cells differentiated into cortical neurons. To confirm the gene’s effect, the researchers engineered mice to lack BCL6, which subsequently resulted in smaller, nerve-diminished brains. The study is published in Nature Neuroscience.
Cell mosaicism—genetic variations between individual cells of a particular type—may account for genomic “instability” observed in induced pluripotent stem cell lines. Researchers led by Mark Gerstein and Flora M. Vaccarino sequenced the complete genomes of stem cells derived from fibroblasts, and found significant copy number variations (CNVs), which are unique genetic variants harbored in DNA. Consequently, these results beg the question of how diverse our own, individual cells are.