Stem Cells in Modeling Human Genetic Diseases

Modeling Fragile X Syndrome in Human Pluripotent Cells.- Induced Pluripotent Stem Cells in Familial Dilated Cardiomyopathy.- Induced Pluripotent Stem Cells and Amyotrophic Lateral Sclerosis.- IPS Cells and Spinocerebellar Ataxia.- Induced Pluripotent Stem Cells and Vascular Disease.- iPS Cells and Cardiomyopathies.- Cancer Stem Cells and Chemoresistance.- Stem Cells in Autism Spectrum Disorders.

Dr. Mayana Zatz is a professor of Human and Medical Genetics and is currently the director of the Human Genome Research Center and Institute of stem-cells in genetic disorders, at the University of São Paulo. She completed a Ph.D. in Human and Medical Genetics there and was a post-doc in medical genetics at the University of California. Her research in human and medical genetics focuses mainly on the following aspects of neuromuscular disorders: novel genes identification, genotype-phenotype correlations, mechanisms of clinical variability and stem-cells as a tool to understand gene expression and their therapeutic applications. She published about 300 peer-reviewed papers that were cited 8700 times ( h=43). She has been actively involved in ethical aspects related to genome research, genetic testing and political decisions regarding the approval of the Brazilian embryonic stem-cell bill by the Congress in 2005 and by the Supreme Court in 2008.

Dr. O. Keith Okamoto is a professor in the Department of Genetics and Evolutionary Biology at the University of São Paulo (USP). Prior to joining the University, he was a professor of Neurosciences at the Medical School of the Federal University of São Paulo. He earned his Ph.D. in Biochemistry and Molecular Biology at the USP and he conducted research as a postdoctoral fellow in the Department of Molecular and Cellular Biology at Harvard University, where he was also affiliated to the Harvard's Bauer Center for Genomics Research. Dr. Okamoto is a member of the Brazilian Cell Therapy Network, and head of the Translational Genomics Laboratory at USP. His current research focuses on the interplay between stem cell biology and cancer, with emphasis on the genetic and molecular mechanisms inducing a stem cell-like phenotype in cancer cells, their role in tumor heterogeneity and aggressiveness, and the contribution of normal stem cells to tumor development and metastasis. Preclinical therapy studies for cancer and some

"The target audience of this book are students, researchers and clinicians interested in the recent developments in the field. The book is well written and, thanks to the good introductory section in each of the chapters, even non-experts in the field will be able to follow. The figures in the book are well-designed ... . The book gives a good overview about the current state of research in these fields and can therefore be recommended for interested readers." (Christian Schnell, Human Genetics, Vol. 135, 2016)