On July 19, NYSCF Board member Margo Alexander and her husband Bob Alexander opened their home to the most advanced stem cell science, hosting NYSCF's Annual Summer Cocktail Reception. This exciting evening included an address by Susan L. Solomon, research descriptions from NYSCF scientists, and a talk on the NYSCF Global Stem Cell Array by Scott Noggle, PhD, Director of the NYSCF Laboratory and the NYSCF - Charles Evans Senior Research Fellow for Alzheimer's Disease.
"The arts and sciences usually exist in two separate worlds, but on Thursday evening The New York Stem Cell Foundation united the two at its first spring benefit at the newly renovated New York Genome Center in SoHo.
"Representing art, Dorothy Lichtenstein, Michael Joaquin Grey, Yigal Ozeri and Donald Sultan sat on a panel alongside NYSCF scientists Valentina Fossati and Scott Noggle."
On June 19, the New York Times ran an article on all too common situation in drug research and development: an experimental drug that had a profound effect on some was discontinued after failing to meet its clinical goals. This trial assessed the alleviation of extreme behavioral symptoms in children with an incurable genetic disorder known as fragile X syndrome.
In a first for the field, NYSCF scientists with Columbia University's Naomi Berrie Diabetes Center researchers have generated patient-specific beta cells, or insulin-producing cells, that accurately reflect a genetic form of diabetes known as maturity onset diabetes of the young (MODY). This incurable disease, caused by a genetic mutation, is characterized by reduced insulin response to sugar, leading to chronic elevated blood sugar levels and increased risk of vascular complications. Through advanced stem cell techniques, the NYSCF scientists took MODY patients’ skin cells and “differentiated”—or reverted—these cells into an embryonic like state. They then corrected the responsible mutation and artificially controlled the maturation of these cells into beta cells progenitors. As a necessary proof-of-concept, they transplanted these stem cell-derived beta cells into mice, which restored insulin levels to those seen in healthy patients. This promising strategy, published in the Journal of Clinical Investigation, may inform therapies for other types of diabetes.
In the June 2013 issue of Current Molecular Medicine, Giuseppe Maria de Peppo, PhD, a NYSCF Research Fellow, co-wrote a review article, assessing the utility of different human progenitor cells, including stem cells, to engineer bone tissue. Currently, bone injuries and conegnital defects are treated with cadaverous bone or artificial substitutes, neither of which fully integrate with surrounding tissue or functionally repair. As an alternative, stem cells carry the potential to become customizable bone tissue. Examining embryonic stem cells, bone marrow cells, and induced pluripotent stem (iPS) cells, the authors conclude that iPS cells, derived from a patient's own skin samples, present the greatest potential to create fully functional, immune-matched bone substitutes.
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.
Multiple Sclerosis Discovery Forum followed NYSCF -- Helmsley Investigator Valentina Fossati, PhD, on her quest that is as scientific as personal. When she turned 30, Fossati was diagnosed with multiple sclerosis (MS), an intreactable neurological disease. She shifted the focus of her stem cell research accordingly, with the goal to find better treatments and even a cure for MS.
As stem cells mature into other cell types, a process known as differentiation, they undergo key molecular-level changes. To understand the regulatory mechanisms that govern this transition, 2012 NYSCF -- Robertson Stem Cell Investgator Alexander Meissner, PhD, Broad Institute of MIT and Harvard, studied a human model of development. In a new Cell study, Meissner profiled embryonic stem cells as they became one of the three germ layers, an initial event before further development into specialized cell types. His team's data pave the way for more faithful processes to procure specialized cell types from stem cells.