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Patient-Specific Induced Pluripotent Stem Cells for the Study of Neurological Diseases

Patient-Specific Induced Pluripotent Stem Cells for the Study of Neurological Diseases
Reported by
Alan Dove

Posted March 29, 2012

Presented By


Induced pluripotent stem cells (iPSCs) are derived from adult somatic cells that, through genetic manipulation, have been reprogrammed to resemble embryonic pluripotent stem cells. Pluripotent stem cells have the unique property of unlimited proliferation in the undifferentiated state while they retain the ability to differentiate into terminal cell types, including neurons, when cultured appropriately. iPSCs hold enormous potential for studying human development and disease, creating new systems to identify promising drugs, and generating customized replacement cells that can be used as therapies directly.

Not only do patient-derived induced pluripotent stem cells circumvent the ethical issues surrounding the derivation of stem cells from human embryos, they have the added benefit of recapitulating the specific neuronal defect in the individual from which they are derived. While the technology for deriving these cells has its own challenges, and new ethical concerns arise around the subject of patient consent, researchers are optimistic about the future of the field.

On December 16, 2011, the New York Academy of Sciences presented a Hot Topics in Life Sciences symposium titled Patient-Specific Induced Pluripotent Stem Cells for the Study of Neurological Diseases. Researchers discussed drug screens using iPSCs from patients with autism or with Parkinson's disease, use of the cell lines to understand how errors in imprinting affect neuronal development and to understand how neuronal defects arise in schizophrenia.

Use the tabs above to find a meeting report and multimedia from this event.

Presentations available from:
Marc Lalande, PhD (University of Connecticut School of Medicine)
Sergiu P. Pasca, MD (Stanford University School of Medicine)


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Journal Articles

Ole Isacson

Deleidi M, Cooper O, Hargus G, et al. Oct4-induced reprogramming is required for adult brain neural stem cell differentiation into midbrain dopaminergic neurons. PLoS ONE 2011;6(5):e19926.

Cooper O, Hallett P, Isacson O. Using stem cells and iPS cells to discover new treatments for Parkinson's disease. Parkinsonism Relat. Disord. 2012;18 Suppl 1:S14-16.

McLean JR, Hallett PJ, Cooper O, et al. Transcript expression levels of full-length alpha-synuclein and its three alternatively spliced variants in Parkinson's disease brain regions and in a transgenic mouse model of alpha-synuclein overexpression. Molecular and Cellular Neurosciences 2011;49(2):230-239.

Marc Lalande

Leung KN, Chamberlain SJ, Lalande M, et al. Neuronal chromatin dynamics of imprinting in development and disease. J. Cell. Biochem. 2011;112(2):365-373.

Martins-Taylor K, Nisler BS, Taapken SM, et al. Recurrent copy number variations in human induced pluripotent stem cells. Nat. Biotechnol. 2011;29(6):488-491.

Martins-Taylor K, Schroeder DI, Lasalle JM, et al. Role of DNMT3B in the regulation of early neural and neural crest specifiers. Epigenetics: Official Journal of the DNA Methylation Society 2012;7(1).

Sergiu Pasca

Pasca SP, Dronca E, Kaucsár T, et al. One carbon metabolism disturbances and the C677T MTHFR gene polymorphism in children with autism spectrum disorders. J. Cell. Mol. Med. 2009;13(10):4229-4238.

Pasca SP, Portmann T, Voineagu I, et al. Using iPSC-derived neurons to uncover cellular phenotypes associated with Timothy syndrome. Nat. Med. 2011;17(12):1657-1662.

Pasca SP, Singer W, Nikolic D. Surround modulation of neuronal responses in V1 is as stable over time as responses to direct stimulation of receptive fields. Cortex 2010;46(9):1199-1203.

Hongjun Song

Bonaguidi MA, Wheeler MA, Shapiro JS, et al. In vivo clonal analysis reveals self-renewing and multipotent adult neural stem cell characteristics. Cell 2011;145(7):1142-1155.

Guo JU, Ma DK, Mo H, et al. Neuronal activity modifies the DNA methylation landscape in the adult brain. Nat. Neurosci. 2011;14(10):1345-1351.

Ming G-L, Song H. Adult neurogenesis in the mammalian brain: significant answers and significant questions. Neuron 2011;70(4):687-702.

Lorenz Studer

Kriks S, Shim J-W, Piao J, et al. Dopamine neurons derived from human ES cells efficiently engraft in animal models of Parkinson's disease. Nature 2011;480(7378):547-551.

Lipchina I, Elkabetz Y, Hafner M, et al. Genome-wide identification of microRNA targets in human ES cells reveals a role for miR-302 in modulating BMP response. Genes Dev. 2011;25(20):2173-2186.

Ming G-L, Brüstle O, Muotri A, et al. Cellular reprogramming: recent advances in modeling neurological diseases. J. Neurosci. 2011;31(45):16070-16075.


Susan DeLaura

Cellular Dynamics International, Inc.
e-mail | publications

Susan DeLaura is responsible for the product management of human induced pluripotent stem cell-derived neurons and endothelial cells at Cellular Dynamics International, Inc (CDI). She brings more than 13 years of experience in the life science industry to CDI. Prior to joining CDI, she served as a Product Manager within EMD Chemicals for the Bioscience business unit. She was responsible strategic and tactical marketing and portfolio management of the Novagen brand flagship products. Additionally, she served as the Manager of Technical Services while at EMD. DeLaura's professional background also includes research in molecular and cell biology in the Department of Human Oncology at the University of Wisconsin-Madison, Comprehensive Cancer Center. While there, she studied the involvement of 4-aminobiphenol (4-ABP) and its derivatives in bladder cancer.

Sandra Engle, PhD

Pfizer, Inc.
e-mail | publications

Sandra Engle is a Senior Principal Scientist in the Pluripotent Stem Cell Biology Laboratory of the Primary Pharmacology Group within Pfizer, Inc. She received her BA in biology with an emphasis in human genetics from Ball State University in Muncie, Indiana. Sandra began her research career as an undergraduate studying T-cell response to cancer in the laboratory of M. Rita Young at the Indiana University Center for Medical Education. She obtained her PhD in Medical and Molecular Genetics from Indiana University School of Medicine in Indianapolis, Indiana, under the direction of Jay Tischfield, for developing a knock-out mouse model of human APRT deficiency. She continued her interest in mouse models with post-doctoral fellowships at the University of Cincinnati College of Medicine in the laboratories of Nelson Horseman studying prolactin deficiency and signaling and of Tom Doetschman studying transforming growth factor beta biology.

Engle moved to pharmaceutical research in 2001 with a position in the Genetically Engineered Mouse Models Group in Aventis (now Sanofi-Aventis) where she applied her skills to generating in vivo mouse knock-out models and in vitro mouse stem cell-derived models. In 2004, she joined the Genetically Modified Models Research Center of Emphasis with Pfizer where she continued to generate in vivo and in vitro models. Currently, she leads the Pluripotent Stem Cell Biology Laboratory within Pfizer's Primary Pharmacology Group which focuses on the generation of human induced pluripotent stem cells, in vitro differentiation of stem cells to terminally differentiated cell types of interest, and the genetic modification of human stem cells.

Mercedes Beyna, MS

Pfizer, Inc.
e-mail | website | publications

Mercedes Beyna is currently a research scientist at Pfizer, where she is using molecular, cellular, genetic, and imaging approaches in the quest to understand the biology underlying autism spectrum disorders. Captivated by neuroscience, she has worked in the field for over 10 years, in both academic and industrial laboratory settings. Beyna attended Binghamton University, earning her undergraduate degree in Biology, and subsequently received her Master's Degree in Biology from New York University. As an active member of the Biochemical Pharmacology Discussion Group, she enjoys developing interesting and educational symposia.

Ken Jones, PhD

Biochemical Pharmacology Discussion Group

Ken Jones received his PhD in Physiology at Rutgers University studying neuronal networks that control behaviors of model organisms. During postdoctoral training at Harvard Medical School with Robert Baughman he developed mammalian primary cell culture techniques to map NMDA and AMPA receptors at synaptic and extrasynaptic sites. His subsequent research in biotech and pharmaceutical companies has provided a number of promising novel drug targets for psychiatric and neurological disorders. At Synaptic Pharmaceutical Corp he co-discovered the heterodimeric nature of GABA B receptors, a new photoreceptor that regulates circadian rhythms, as well as several novel hypothalamic neuropeptide receptors. In his most recent position at Lundbeck Research (Paramus, NJ) he was responsible for capital and process improvements that dramatically improved assay throughput in the HTS group, and he served leadership roles in a handful of early- to late-stage drug discovery projects. He has enjoyed co-organizing a variety of Academy symposia under the auspices of the Biochemical Pharmacology Discussion Group.

Jennifer Henry, PhD

The New York Academy of Sciences

Jennifer Henry received her PhD in plant molecular biology from the University of Melbourne, Australia, with Paul Taylor at the University of Melbourne and Phil Larkin at CSIRO Plant Industry in Canberra, specializing in the genetic engineering of transgenic crops. She was then appointed as Associate Editor, then Editor, of Functional Plant Biology at CSIRO Publishing. She moved to New York for her appointment as a Publishing Manager in the Academic Journals division at Nature Publishing Group, where she was responsible for the publication of biomedical journals in nephrology, clinical pharmacology, hypertension, dermatology, and oncology. Henry joined the Academy in 2009 as Director of Life Sciences and organizes 35 – 40 seminars each year. She is responsible for developing scientific content in coordination with the various life sciences Discussion Group steering committees, under the auspices of the Academy's Frontiers of Science program. She also generates alliances with outside organizations interested in the programmatic content.


Ole Isacson, MD

McLean Hospital and Harvard Medical School
e-mail | website | publications

Ole Isacson is Professor of Neurology (Neuroscience) at Harvard Medical School. He is the Director and Chair of the Neuroregeneration Research Institute at McLean Hospital. The Neuroregeneration Institute focuses on advancing conceptually novel therapies for Parkinson's disease, Alzheimer's disease, Huntington's disease, Amyotrophic Lateral Sclerosis, and related neurodegenerative or neuropsychiatric diseases. Its goal is to provide cutting-edge research that can be translated into relevant therapeutic gains for patients in need. The Institute works with other research teams, governmental agencies, and pharmaceutical companies to maximize the therapeutic benefits of this research. Isacson is also Principal Faculty of the Harvard Stem Cell Institute and a member of the Scientific Advisory Boards for the Harvard NeuroDiscovery Center and the Michael J. Fox Foundation. He is the past Receiving Editor of the European Journal of Neuroscience, current Editor-in-Chief of Molecular and Cellular Neuroscience, and a member of many other editorial boards. Isacson has published over 280 original peer-reviewed scientific articles and reviews in his fields, and he, his laboratory, and collaborators have received several awards for this work.

Marc Lalande, PhD

University of Connecticut Health Center
e-mail | website | publications

After receiving a PhD in Medical Biophysics from the University of Toronto, Marc Lalande pursued postdoctoral training in the Department of Pediatrics, Harvard Medical School and Children's Hospital, Boston, MA. He was Assistant Professor, Department of Pathology and Center for Human Genetics, McGill University, Montreal, Québec before returning to Boston Children's Hospital. He remained at Harvard Medical School as an Associate Professor of Pediatrics and an Assistant Investigator of the Howard Hughes Medical Institute until 1998. He is currently Physicians Health Services Professor and Chairman of the Department of Genetics Developmental Biology and Director of the University of Connecticut Stem Cell Institute. His area of expertise is human molecular genetics and genomic imprinting.

Sergiu P. Pasca, MD

Stanford University School of Medicine
e-mail | website | publications

Sergiu Pasca is a postdoctoral research fellow in the Department of Neurobiology at Stanford University. Pasca has a long-standing interest in understanding mechanisms underlying neurodevelopmental disorders. During his medical studies in Romania he worked in the laboratory of Maria Dronca towards understanding metabolic disturbances in children with autism spectrum disorders. During this time, Pasca also spent time training in neurophysiology of the visual cortex in Danko Nikolic's lab at the Max Planck Institute for Brain Research in Germany. In 2009, he joined the laboratory of Ricardo Dolmetsch at Stanford University, where he is using the induced pluripotent stem cell (iPSC) technology to identify cellular endophenotypes for neuropsychiatric diseases.

Hongjun Song, PhD

Johns Hopkins University
e-mail | website | publications

Hongjun Song is a Professor in the Departments of Neurology and Neuroscience, Director of the Stem Cell Biology Program in the Institute for Cell Engineering at the Johns Hopkins University School of Medicine. Song is a leader in the research of adult neural stem cells and neurogenesis. He has published many high-profile papers from his laboratory, including in Nature, Science, and Cell, as well as many influential reviews in Annual Reviews of Neuroscience and Current Opinion in Neurobiology. Song has won several awards including the Klingenstein Fellowship Awards in the Neuroscience (2003), the McKnight Scholar Award (2006), the Inaugural Young Investigator Award of the Chinese Biological Investigators Society (2008), the NARSAD Independent Investigator Award (2008), and the Rising Star Award from International Mental Health Research Organization (2009). He was honored in 2008 with Young Investigator Award from the Society for Neuroscience. He is a member of the Faculty of 1000 Biology Neurobiology of Disease and Regeneration Section of the Neuroscience Faculty and on the editorial board for several journals.

Song's laboratory has pioneered using the "single-cell genetics" approach to examine development of new neurons from neural stem cells in adult animals in combination with state-of-the-art technologies in confocal and electron microscopy, multiphoton confocal imaging, and electrophysiology. His laboratory also established the methodology to derive patient-specific induced pluripotent stem cells to model human neurological diseases, to understand disease mechanisms and to screen drugs.

Lorenz Studer, MD

Memorial Sloan-Kettering Cancer Center
e-mail | website | publications

A native of Switzerland, Lorenz Studer graduated from medical school in 1991 and received his doctoral degree in neuroscience at the University of Bern in 1994. While there, he initiated studies with Christian Spenger, leading to the first clinical trial of fetal tissue transplantation for Parkinson's disease in Switzerland in December 1995. Studer next pursued his research interests at the National Institutes of Health (NIH) in Bethesda, Maryland, where he worked in the laboratory of Ronald D. McKay. At the NIH he pioneered techniques that allow the generation of dopamine cells in culture from dividing precursor cells. In 1998, he was first to demonstrate that the transplantation of dopamine cells generated in culture improve clinical symptoms in Parkinson's-like rats.

In 2000, he moved to New York City where he started his own research program at the Memorial Sloan-Kettering Cancer Center with a focus on stem cells and brain repair. Major early contributions of his lab were the in vitro derivation of midbrain dopamine neurons from embryonic stem cells, mouse nuclear transfer embryonic stem cells and a novel type of pluripotent parthenogenetic stem cell in monkey. His laboratory was also first to demonstrate "therapeutic cloning" in a mouse model of a CNS disorder, and he has pioneered studies on the directed differentiation, high-throughput screening and genetic modification of human ES cells. His most recent work increasingly focuses on the biology of human ES cells and human induced pluripotent stem cells developing novel strategies at the interface of developmental biology, regenerative medicine, and disease modeling.Studer is the founding director of the Sloan-Kettering Center for Stem Cell Biology, a Member in the Developmental Biology Program and the Department of Neurosurgery, and Professor in Neuroscience at Weill-Cornell Graduate School. He also currently heads the steering committee of the Tri-institutional stem cell initiative (Sloan-Kettering Institute, Weill-Cornell Medical School, and Rockefeller University).

Alan Dove, PhD

Alan Dove is a science writer and reporter for Nature Medicine, Nature Biotechnology, and Bioscience Technology. He also teaches at the NYU School of Journalism, and blogs at


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