The New York Stem Cell Foundation

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.

Read an article on this advance in Forbes >>

Read more in Bloomberg Businessweek >>

Read about this work in Time >>

Read a post on the importance of this advance on the CIRM blog >>

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. 

Read the full article on Multiple Sclerosis Discovery Forum >>

Read more about Dr. Fossati's research at NYSCF >>

In a step towards personalized bone grafts to treat traumatic injury or congenital defects, a study, led by Darja Marolt, PhD, and Giuseppe Maria de Peppo, PhD, and published in the Proceedings of the National Academy of Sciences, reports the generation of patient-specific bone substitutes from skin cells for repair of large bone defects. This advance will facilitate the development of customizable, three-dimensional bone grafts on-demand, matched to fit the exact needs and immune profile of a patient. Taking skin cells, the NYSCF scientists utilized an advanced technique called “reprogramming” to revert adult cells into an embryonic-like state. The researchers differentiated the resultant induced pluripotent stem (iPS) cells into bone-forming progenitors and then seeded these cells onto a three-dimensional scaffold, which was placed into an artificial, biologically active environment, a bioreactor. Analysis revealed that, even in vitro, bone formed; and, when implanted in mice, the bone matured into typical dense, mineral-rich tissue. Next steps for this proof-of-concept research include protocol optimization and long-term safety trials in animal models.

Read an article on this breakthrough in the Wall Street Journal >>

Find a PDF version of the Wall Street Journal Article >>

Read a press release on this research >>

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.

Read this study in Cell >>

Reprogramming, or reverting, adult cells into an embryonic-like state is a dynamic process: some cells retain their adult cell features whereas others become fully pluripotent, indicating that they can become other cell types. Traditional assays to identify pluripotent stem cells range from observation of cell morphology to implanting these cells into mouse embryos to assess developmental contribution. Recent advances in bioinformatics and gene expression profiling have led to a new generation of pluripotency assays. In this Stem Cell Research and Therapy review article, NYSCF scientists describe these new technologies, ultimately calling for a standardized approach. More reliable measures for pluripotency lend power to stem cell-based disease investigations and scalability to the development of potential cell therapies.

Read the review article here >>

The brain, dense with neurons, translates external stimuli into behavioral responses and learned memories, and just how this information is decoded can mean the difference between life and death. Focusing on a model system of a housefly's response to odors, 2012 NYSCF - Robertson Investigator Vanessa Ruta, PhD, of The Rockefeller University traces the anatomical connections between different neuron types in a new Nature study. Following the path of neurons responsible for receiving odor inputs to those that sync up in the mushroom body, a fly's brain center where odors are decoded, Ruta with colleagues found highly random connections. Instead of one input connecting to one output, odors were translated down a multivariate path. This highly plastic configuration may be to the fly's advantage: it can respond appropriately to a full range of unpredictable situations and odors. Ultimately, Ruta aims to use these insights to identify strategies that counter the spread of insect-borne disease.

Read more on Dr. Ruta's study >>

The New York Stem Cell Foundation (NYSCF) was a principal supporter of a new study published today in Cell Stem Cell that employed a novel stem cell-based screening method to identify a promising chemical compound for Lou Gehrig’s disease, or amyotrophic lateral sclerosis (ALS). NYSCF Scientific Advisor and Medical Advisory Board Member Lee Rubin, PhD, Harvard Stem Cell Institute (HSCI) Principal Faculty member, led this study. Using a new stem cell-based drug screening technology with the potential to reinvent and greatly reduce the cost of the way new pharmaceuticals are developed, Rubin’s team has found a compound more effective in protecting the neurons killed in ALS than two drugs that failed in human clinical trials after hundreds of millions of dollars had been invested in them.

Read the full press release >>

At our annual Innovator's retreat, our 2010 NYSCF -- Robertson Stem Cell Investigators Paul Tesar, PhD, of Case Western University, and Marius Wernig, MD, PhD, of Stanford University, discovered that they were working on the same topic, the direct conversion of skin cells into myelin-producing cells, called oligodendrocytes. That conversation led to the back-to-back publication of two studies in Nature Biotechnology. We spoke with Marius and Paul on how they worked together to publish this exciting discovery that could inform the treatment of multiple sclerosis and other myelin disorders. (Continued...)

For his achievements, 2010 -- NYSCF Robertson Stem Cell Investigator Marius Wernig of Stanford University is the recipient of the 2013 ISSCR-University of Pittsburg Outstanding Young Investigator award. Wernig's research into the direct reprogramming of adult cells into other cell types has catalyzed the entire field. He will be honored in ceremony at the ISSCR 11th Annual Meeting in June.

Read more about the ISSCR award >>