News Article | October 26, 2016
New York, NY (October 25, 2016) - The New York Stem Cell Foundation (NYSCF) announced today that Feng Zhang, PhD, is the 2016 recipient of the NYSCF - Robertson Stem Cell Prize for his pioneering advances to edit human and plant genomes using CRISPR-Cas9. "We are particularly pleased to recognize Feng with the NYSCF - Robertson Stem Cell Prize," explained Susan L. Solomon, CEO and Co-founder of NYSCF. "A 2014 NYSCF - Robertson Stem Cell Investigator and member of our outstanding Innovator community, his work represents a new frontier in research that has already dramatically changed the scientific and medical landscape, ushering in new treatments and therapies that have never before been possible." Dr. Zhang is an Associate Professor of Neuroscience and Biological Engineering at the Massachusetts Institute of Technology (MIT), a Core Member of the Broad Institute of MIT and Harvard, an Investigator at the McGovern Institute for Brain Research at MIT, and the W.M. Keck Career Development Professor in Biomedical Engineering in the Departments of Brain and Cognitive Sciences and Biological Engineering at MIT. His development of the CRISPR-Cas9 gene editing system and seminal 2013 Science paper where he described successful gene editing using the technique in human cells opened an entirely new arm of scientific research and inquiry. Critically, the CRISPR-Cas9 system and later advances, also developed by Zhang, are easy to execute with almost endless possibility for new research enabling scientists to change, delete and replace any gene of any animal. This system has unquestionably accelerated research around the world that will benefit human health. "It is really an honor for me and everyone in my team to be recognized for the work we are doing, and I am excited to apply the genome editing tools that we have developed to study complex human diseases," said Dr. Zhang. "This prize will further our work to develop and apply molecular tools to identify genetic variants involved in disease phenotypes and refining these tools for therapeutic use." Previously, Dr. Zhang has received numerous awards and honors, including the NIH Director's Pioneer Award, the Popular Science Brilliant 10 Award, named one of Nature's "10 people who mattered in 2013," The Society for Neuroscience Young Investigator Award, and recently, the Canada Gairdner International Award and the Tang Prize. "Gene editing using RNA guided endonucleases has opened entirely new frontiers for regenerative medicine, and Dr. Zhang's pioneering work in this area has ignited a new era of discovery that will transform the way we study and treat human disease," said Amy Wagers, PhD, 2013 NYSCF - Robertson Stem Cell Prize recipient and NYSCF - Robertson Stem Cell Investigator Awards Jury member from Harvard University. "I am so happy to see him recognized by the 2016 NYSCF - Roberson Stem Cell Prize, acknowledging the profound impact of his innovation and insight for scientists and patients around the globe." The NYSCF - Robertson Stem Cell Prize has been awarded annually since 2011 to an outstanding young stem cell scientist in recognition of significant and path breaking translational stem cell research. All NYSCF - Robertson Stem Cell Prize recipients receive $200,000 to be used for research purposes at their discretion. The New York Stem Cell Foundation (NYSCF) Research Institute is an independent organization accelerating cures and better treatments for patients through stem cell research. The NYSCF global community includes over 140 researchers at leading institutions worldwide, including the NYSCF - Druckenmiller Fellows, the NYSCF - Robertson Investigators, the NYSCF - Robertson Stem Cell Prize Recipients, and NYSCF Research Institute scientists and engineers. The NYSCF Research Institute employs over 45 researchers in New York, and is an acknowledged world leader in stem cell research and in developing pioneering stem cell technologies, including the NYSCF Global Stem Cell ArrayTM. NYSCF focuses on translational research in a model designed to overcome the barriers that slow discovery and replace silos with collaboration. For more information, visit http://www.
News Article | October 26, 2016
NEW YORK, NY (October 25, 2016) - The New York Stem Cell Foundation (NYSCF) announced the 2016 class of NYSCF - Robertson Investigators, welcoming six of the most talented stem cell researchers and neuroscientists from around the world into the NYSCF Investigator Program. The NYSCF Investigator Program fosters and encourages promising early career scientists whose cutting-edge research holds the potential to accelerate treatments and cures, and provides support for the NYSCF - Robertson Stem Cell Investigator Awards and the NYSCF - Robertson Neuroscience Investigator Awards. The awards provide critical seed funding - $1.5 million over five years - to outstanding young scientists as they move beyond their postdoctoral training to establish their own, independent laboratories. This year, three scientists joined the seventh class of NYSCF - Robertson Stem Cell Investigators and three others joined the sixth class of NYSCF - Robertson Neuroscience Investigators. "These six outstanding researchers focus on the most promising, translational research and we are pleased to welcome them into our global NYSCF Innovator community," said Susan L. Solomon, CEO and Co-founder of NYSCF. "Enabling their important research as they move into the next phase of their careers is a key priority of our mission, and their work will unquestionably accelerate progress towards cures for the entire field." These awards enable Investigators to pursue high-risk/high-reward research that traditional funding does not support. To date, the NYSCF global community includes 41 NYSCF - Robertson Investigators and Alumni at 32 institutions throughout the world. "The NYSCF Investigator Programs are a critical part of encouraging promising young scientists to pursue innovative stem cell and neuroscience research as a career," stated Leslie Vosshall, PhD, Investigator at the Howard Hughes Medical Institute and Robin Chemers Neustein Professor in the Laboratory of Neurogenetics and Behavior at The Rockefeller University and NYSCF - Robertson Neuroscience Awards jury member. "The award winners are creative, out-of-the-box thinkers pursuing high-risk/high-reward research that pushes the boundaries of basic research, and in many cases yielding translational results with near-term impact in the clinic." Catherine Dulac, PhD, Harvard University, chaired the NYSCF - Robertson Neuroscience Investigator Awards selection committee and was joined on the jury by Jonathan Flint, MD, Wellcome Trust Centre for Human Genetics and University of California, Los Angeles; Arnold Kriegstein, MD, PhD, University of California, San Francisco; and Dr. Vosshall. The NYSCF - Robertson Stem Cell Investigator Awards selection committee included 2015 MacArthur Fellow Lorenz Studer, MD, Memorial Sloan-Kettering Cancer Center; Fiona Watt, DPhil, King's College London in the United Kingdom; 2013 NYSCF - Robertson Stem Cell Prize recipient Amy Wagers, PhD, Harvard University; Owen Witte, MD, University of California, Los Angeles; and Irv Weissman, MD, Stanford University. The New York Stem Cell Foundation (NYSCF) Research Institute is an independent organization accelerating cures and better treatments for patients through stem cell research. The NYSCF global community includes over 140 researchers at leading institutions worldwide, including the NYSCF - Druckenmiller Fellows, the NYSCF - Robertson Investigators, the NYSCF - Robertson Stem Cell Prize Recipients, and NYSCF Research Institute scientists and engineers. The NYSCF Research Institute employs over 45 researchers in New York, and is an acknowledged world leader in stem cell research and in developing pioneering stem cell technologies, including the NYSCF Global Stem Cell ArrayTM. NYSCF focuses on translational research in a model designed to overcome the barriers that slow discovery and replace silos with collaboration. For more information, visit http://www.
News Article | December 12, 2016
NEW YORK NY (December 12, 2016)--Columbia University Medical Center (CUMC) researchers have discovered that a deficiency of the enzyme prohormone covertase (PC1) in the brain is linked to most of the neuro-hormonal abnormalities in Prader-Willi syndrome, a genetic condition that causes extreme hunger and severe obesity beginning in childhood. The discovery provides insight into the molecular mechanisms underlying the syndrome and highlights a novel target for drug therapy. The findings were published online today in the Journal of Clinical Investigation. "While we've known for some time which genes are implicated in Prader-Willi syndrome, it has not been clear how those mutations actually trigger the disease," said lead author Lisa C. Burnett, PhD, a post-doctoral research scientist in pediatrics at CUMC. "Now that we have found a key link between these mutations and the syndrome's major hormonal features, we can begin to search for new, more precisely targeted therapies." An estimated one in 15,000 people have Prader-Willi syndrome (PWS). The syndrome is caused by abnormalities in a small region of chromosome 15, which leads to dysfunction in the hypothalamus--which contains cells that regulate hunger and satiety--and other regions of the brain. A defining characteristic of PWS is insatiable hunger. People with PWS typically have extreme obesity, reduced growth hormone and insulin levels, excessive levels of ghrelin (a hormone that triggers hunger), and developmental disabilities. There is no cure and few effective treatments for PWS. Dr. Burnett and her colleagues used stem cell techniques to convert skin cells from PWS patients and unaffected controls into brain cells. Analysis of the stem cell-derived neurons revealed significantly reduced levels of PC1 in the patients' cells, compared to the controls. The cells from PWS patients also had abnormally low levels of a protein, NHLH2, which is made by NHLH2, a gene that also helps to produce PC1. To confirm whether PC1 deficiency plays a role in PWS, the researchers examined transgenic mice that do not express Snord116, a gene that is deleted in the region of chromosome 15 that is associated with PWS. The mice were found to be deficient in NHLH2 and PC1 and displayed most of the hormone-related abnormalities seen in PWS, according to study leader Rudolph L. Leibel, MD, professor of pediatrics and medicine and co-director of the Naomi Berrie Diabetes Center at CUMC. "The findings strongly suggest that PC1 is a good therapeutic target for PWS," said Dr. Burnett. "There doesn't seem to be anything wrong with the gene that makes PC1--it's just not getting activated properly. If we could elevate levels of PC1 using drugs, we might be able to alleviate some of the symptoms of the syndrome." "This is an outstanding example how research on human stem cells can lead to novel insight into a disease and provide a platform for the testing of new therapies," said Dieter Egli, PhD, a stem cell scientist who is an assistant professor of developmental cell biology (in Pediatrics) and a senior author on the paper. "This study changes how we think about this devastating disorder," said Theresa Strong, PhD, chair of the scientific advisory board of the Foundation for Prader-Willi Research and the mother of a child with PWS. "The symptoms of PWS have been very confusing and hard to reconcile. Now that we have an explanation for the wide array of symptoms, we can move forward with developing a drug that addresses their underlying cause, instead of treating each symptom individually." Following the findings reported in this paper, the Columbia research team began collaborating with Levo Therapeutics, a PWS-focused biotechnology company, to translate the current research into therapeutics. The study is titled, "Deficiency in prohormone convertase PC1 impairs prohormone processing in Prader-Willi syndrome." The other contributors are: Charles A. LeDuc (CUMC), Carlos R. Sulsona (University of Florida College of Medicine Gainesville, FL), Daniel Paull (New York Stem Cell Foundation Research Institute, New York, NY), Richard Rausch (CUMC), Sanaa Eddiry (Université Paul Sabatier, Toulouse, France), Jayne F. Martin Carli (CUMC), Michael V. Morabito (CUMC), Alicja A. Skowronski (CUMC), Gabriela Hubner (Packer Collegiate Institute), Matthew Zimmer (New York Stem Cell Foundation Research Institute), Liheng Wang (CUMC), Robert Day (Université de Sherbrooke, Quebec, Canada), Brynn Levy (CUMC), Ilene Fennoy (CUMC), Beatrice Dubern (Sorbonne University, University Pierre et Marie-Curie, Paris, France), Christine Poitou (Sorbonne University), Karine Clement (Sorbonne University), Merlin G. Butler (Kansas University Medical Center, Kansas City, KS), Michael Rosenbaum (CUMC), Jean Pierre Salles (Université de Toulouse. Toulouse, France), Maithe Tauber (Université de Toulouse), Daniel J. Driscoll (University of Florida College of Medicine), and Dieter Egli (CUMC and New York Stem Cell Foundation Research Institute). The study was supported by grants from the Foundation for Prader-Willi Research, Russell Berrie Foundation, Rudin Foundation, The New York Stem Cell Foundation, Helmsley Foundation, and National Institutes of Health (RO1DK52431 and P30 DK26687). The authors declare no conflicts of interest. Columbia University Medical Center provides international leadership in basic, preclinical, and clinical research; medical and health sciences education; and patient care. The medical center trains future leaders and includes the dedicated work of many physicians, scientists, public health professionals, dentists, and nurses at the College of Physicians and Surgeons, the Mailman School of Public Health, the College of Dental Medicine, the School of Nursing, the biomedical departments of the Graduate School of Arts and Sciences, and allied research centers and institutions. Columbia University Medical Center is home to the largest medical research enterprise in New York City and State and one of the largest faculty medical practices in the Northeast. The campus that Columbia University Medical Center shares with its hospital partner, NewYork-Presbyterian, is now called the Columbia University Irving Medical Center. For more information, visit cumc.columbia.edu or columbiadoctors.org.
PubMed | Baylor College of Medicine, McLean Hospital, Hoffmann-La Roche, Cold Spring Harbor Laboratory and 4 more.
Type: | Journal: NPJ schizophrenia | Year: 2016
Neurodevelopmental disorders, such as autism spectrum disorders (ASD) and schizophrenia (SZ), are complex disorders with a high degree of heritability. Genetic studies have identified several candidate genes associated with these disorders, including contactin-associated protein-like 2 (
News Article | March 16, 2016
The stem cells described in this paper are the first human cells that are known to be capable of cell division with just one copy of the parent cell's genome. Human cells are considered 'diploid' because they inherit two sets of chromosomes, 46 in total, 23 from the mother and 23 from the father. The only exceptions are reproductive (egg and sperm) cells, known as 'haploid' cells because they contain a single set of 23 chromosomes. These haploid cells cannot divide to make more eggs and sperm. Previous efforts to generate embryonic stem cells using human egg cells had resulted in diploid stem cells. In this study, the scientists triggered unfertilized human egg cells into dividing. They then highlighted the DNA with a fluorescent dye and isolated the haploid stem cells, which were scattered among the more populous diploid cells. The researchers showed that these haploid stem cells were pluripotent—meaning they were able to differentiate into many other cell types, including nerve, heart, and pancreatic cells—while retaining a single set of chromosomes. "This study has given us a new type of human stem cell that will have an important impact on human genetic and medical research," said Nissim Benvenisty, MD, PhD, Director of the Azrieli Center for Stem Cells and Genetic Research at the Hebrew University of Jerusalem and principal co-author of the study. "These cells will provide researchers with a novel tool for improving our understanding of human development, and the reasons why we reproduce sexually, instead of from a single parent." The researchers were also able to show that by virtue of having just a single copy of a gene to target, haploid human cells may constitute a powerful tool for genetic screens. Being able to affect single-copy genes in haploid human stem cells has the potential to facilitate genetic analysis in biomedical fields such as cancer research, precision and regenerative medicine. "One of the greatest advantages of using haploid human cells is that it is much easier to edit their genes," explained Ido Sagi, the PhD student who led the research at the Azrieli Center for Stem Cells and Genetic Research at the Hebrew University of Jerusalem. In diploid cells, detecting the biological effects of a single-copy mutation is difficult, because the other copy is normal and serves as "backup." Since the stem cells described in this study were a genetic match to the egg cell donor, they could also be used to develop cell-based therapies for diseases such as blindness, diabetes, or other conditions in which genetically identical cells offer a therapeutic advantage. Because their genetic content is equivalent to germ cells, they might also be useful for reproductive purposes. "This work is an outstanding example of how collaborations between different institutions, on different continents, can solve fundamental problems in biomedicine," said Dieter Egli, PhD, principal co-author of the study, and Assistant Professor of Developmental Cell Biology in Pediatrics at Columbia University Medical Center and a Senior Research Fellow at the NYSCF Research Institute and a NYSCF-Robertson Investigator. The research, supported by The New York Stem Cell Foundation, the New York State Stem Cell Science Program, and by the Azrieli Foundation, underscores the importance of private philanthropy in advancing cutting-edge science. More information: Ido Sagi et al. Derivation and differentiation of haploid human embryonic stem cells, Nature (2016). DOI: 10.1038/nature17408
Tandon N.,Columbia University |
Marolt D.,The New York Stem Cell Foundation |
Cimetta E.,Columbia University |
Vunjak-Novakovic G.,Columbia University
Biotechnology Advances | Year: 2013
Stem cells hold promise to revolutionize modern medicine by the development of new therapies, disease models and drug screening systems. Standard cell culture systems have limited biological relevance because they do not recapitulate the complex 3-dimensional interactions and biophysical cues that characterize the in vivo environment. In this review, we discuss the current advances in engineering stem cell environments using novel biomaterials and bioreactor technologies. We also reflect on the challenges the field is currently facing with regard to the translation of stem cell based therapies into the clinic. © 2013 Elsevier Inc.
The New York Stem Cell Foundation | Date: 2013-12-20
The present invention relates to the diagnosis, prognosis, progression, and treatment of Alzheimers disease. In particular, Alzheimers disease can be characterized by the differential expression of certain genes, including ASB9, BIK, C7orfl6, NDP, NLRP2, PLP1, SLC45A2, TBX2, TUBB4, ZNF300, ADM2, FLJ35024, MT2A, PTGS2, ABCC2, ECEL1, EGFL8, FSTL5, and SMOC1. The present invention also relates to systems and methods of analyzing Alzheimers disease, methods of screening for treatment agents for Alzheimers disease and kits for the analysis of Alzheimers disease.
The New York Stem Cell Foundation | Date: 2014-12-29
In some embodiments, the present invention provides tissue grafts, such as vascularized bone grafts, and methods for preparing and using such tissue grafts. In some embodiments the tissue grafts are made using pluripotent stem cells, such as autologous pluripotent stem cells. In some embodiments, the tissue grafts are made by creating a digital model of a tissue portion to be replaced or repaired, such as a bone defect, partitioning the model into two or more model segments, and then producing tissue graft segments having a size and shape corresponding to that of the model segments. Such tissue graft segments may be assembled to form a tissue graft having a size and shape corresponding to that of the tissue portion to be replaced or repaired.
The New York Stem Cell Foundation | Date: 2011-11-22
Methods are provided for producing a human embryo capable of developing to the blastocyst stage. The method includes transferring a human somatic cell genome into a mature human oocyte by nuclear transfer and activating the oocyte, without removing the oocyte genome. Pluripotent human embryonic stem cells, and methods of obtaining these, are also provided.
The New York Stem Cell Foundation | Date: 2011-06-13
The invention provides improved methods for producing induced pluripotent stem cells (iPSC) from adult fibroblasts. The methods include contacting adult fibroblasts with a reprogramming composition suitable for reprogramming the adult fibroblasts to iPSC, under conditions effective for the reprogramming composition to penetrate the adult fibroblasts, followed by culturing the contacted fibroblasts for a time period sufficient for the cells to be reprogrammed. The cultured cells are then sorted to select cells based upon their expression of the cell membrane surface markers CD13^(NEG )SSEA4^(POS )Tra-1-60^(POS). iPSC colonies are then identified from the sorted cells.