Regenerative Medicine and Cell Therapy
This volume presents an overview of the latest advances in the field of regenerative medicine and cell therapy, spanning topics such as neurological, ocular surface, skin, cardiac, musculoskeletal, liver, and gastrointestinal diseases and diabetes, in add
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Cell Therapy for Neurodegenerative Disorders Ilyas Singec
Abstract The last decade has seen tremendous progress in stem cell biology, targeted genome editing, bioengineering, and systems neuroscience supporting the notion that cell therapy of various disorders of the central nervous system (CNS) may become clinical reality in the near future. In particular, the advent of induced pluripotent stem (iPS) cells and access to large quantities of patient- and diseasespecific cellular material offers unique opportunities for developmental biology and regenerative medicine. It is now possible to investigate the molecular underpinnings of monogenic and complex human diseases using stem cell-derived neural phenotypes. Molecular insights from such studies will leverage the development of diagnostic tools, biomarkers, drugs, and cell replacement with the ultimate goal to halt or reverse the course of devastating maladies. In this book chapter, I shall discuss the opportunities and emerging challenges of stem cellbased therapies and highlight common neurological diseases that may benefit from such iatrogenic interventions.
1.1 Introduction Owing to advances in modern medicine over the last decades, higher life expectancy has increased the proportion of the aged human population in industrialized countries. This profound demographic change allows the prognosis that chronic I. Singec (&) Sanford-Burnham Medical Research Institute, La Jolla, CA, USA e-mail: [email protected] Present Address I. Singec Pfizer Neuroscience, 700 Main Street, Cambridge, MA 02139, USA
H. Baharvand and N. Aghdami (eds.), Regenerative Medicine and Cell Therapy, Stem Cell Biology and Regenerative Medicine, DOI: 10.1007/978-1-62703-098-4_1, Ó Springer Science+Business Media New York 2013
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I. Singec
incurable disorders of the central nervous system (CNS) will have an unprecedented social and economic impact on society and the health care systems. In light of this prospect, regenerative medicine depends on innovative strategies and medical breakthroughs that can directly translate into novel therapeutics. The human brain is characterized by enormous cellular and synaptic complexity and any attempt for repairing or replacing nervous tissue is among the most formidable goals in medicine. Neurons in the human brain are postmitotic and as old as the diseased patient [1]. In general, the human CNS has limited regenerative potential after injury and the pathobiology of neurodegenerative diseases is intricate and difficult to study. In most cases, human samples are derived from postmortem tissue with inherent problems such as poor tissue preservation and lack of standardization. In addition, postmortem specimens often reflect the endstage of a given disease thereby limiting the study of prodromal changes. The clinical manifestations of CNS diseases are determined by the underlying anatomical location of the lesion, the affected cell type(s), the age of onset, genetic background (familial or sporadic), and the environmental context (e.g. toxins, pestic
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