Programmed Cells from Basic Neuroscience to Therapy

1. Front Matter

   Pages i-xii

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2. iPS Cell Technology and Disease Research: Issues To Be Resolved

   • Rudolf Jaenisch

   Pages 1-7

3. Therapeutic Somatic Cell Reprogramming by Nuclear Transfer

   • Stan Wang, John B. Gurdon

   Pages 9-15

4. Induction of Neural Lineages from Mesoderm and Endoderm by Defined Transcription Factors

   • Marius Wernig

   Pages 17-30

5. Proposing a Model for Studying Primate Development Using Induced Pluripotent Stem Cells

   • Maria C. N. Marchetto, Alysson R. Muotri, Fred H. Gage

   Pages 31-39

6. HTT Evolution and Brain Development

   • Chiara Zuccato, Elena Cattaneo

   Pages 41-55

7. Human Pluripotent and Multipotent Stem Cells as Tools for Modeling Neurodegeneration

   • Jerome Mertens, Philipp Koch, Oliver Brüstle

   Pages 57-66

8. Human Stem Cell Approaches to Understanding and Treating Alzheimer’s Disease

   • Lawrence S. B. Goldstein

   Pages 67-73

9. Potential of Stem Cell-Derived Motor Neurons for Modeling Amyotrophic Lateral Sclerosis (ALS)

   • Derek H. Oakley, Gist F. Croft, Hynek Wichterle, Christopher E. Henderson

   Pages 75-91

10. Using Pluripotent Stem Cells to Decipher Mechanisms and Identify Treatments for Diseases That Affect the Brain

    • Marc Peschanski, Cécile Martinat

    Pages 93-99

11. Modeling Autism Spectrum Disorders Using Human Neurons

    • Alysson Renato Muotri

    Pages 101-117

12. On the Search for Reliable Human Aging Models: Understanding Aging by Nuclear Reprogramming

    • Ignacio Sancho-Martinez, Emmanuel Nivet, Juan Carlos Izpisua Belmonte

    Pages 119-130

13. Back Matter

    Pages 131-132

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The recent advances in Programming Somatic Cell (PSC) including induced Pluripotent Stem Cells (iPS) and Induced Neuronal phenotypes (iN), has changed our experimental landscape and opened new possibilities. The advances in PSC have provided an important tool for the study of human neuronal function as well as neurodegenerative and neurodevelopmental diseases in live human neurons in a controlled environment. For example, reprogramming cells from patients with neurological diseases allows the study of molecular pathways particular to specific subtypes of neurons such as dopaminergic neurons in Parkinson’s Disease, Motor neurons for Amyolateral Sclerosis or myelin for Multiple Sclerosis. Detecting disease-specific molecular signatures in live human brain cells, opens possibilities for early intervention therapies and new diagnostic tools. Importantly, once the neurological neural phenotype is detected in vitro, the so-called “disease-in-a-dish” approach allows for the screening of drugs that can ameliorate the disease-specific phenotype. New therapeutic drugs could either act on generalized pathways in all patients or be patient-specific and used in a personalized medicine approach. However, there are a number of pressing issues that need to be addressed and resolved before PSC technology can be extensively used for clinically relevant modeling of neurological diseases. Among these issues are the variability in PSC generation methods, variability between individuals, epigenetic/genetic instability and the ability to obtain disease-relevant subtypes of neurons . Current protocols for differentiating PSC into specific subtypes of neurons are under development, but more and better protocols are needed. Understanding the molecular pathways involved in human neural differentiation will facilitate the development of methods and tools to enrich and monitor the generation of specific subtypes of neurons that would be more relevant in modeling differentneurological diseases.

• Alzheimer's disease
• Huntington's disease
• Neurodegenerative and neurodevelopmental diseases
• Programming Somatic Cell (PSC)
• Rett syndrome
• Stem cells

• , Laboratory of Genetics, The Salk Institute f. Biological Studies, La Jolla, USA

  Fred H. Gage

• Fondation Ipsen, Boulogne Billancourt, France

  Yves Christen

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