One of the biggest challenges faced in medical research had been to create accurate and relevant models of human disease. A number of good animal models have been developed to understand the pathophysiology. However, not all of them reflect the human disorder, a classic case being Usher’s syndrome where the mutant mice do not have the same visual and auditory defects that patients face. There are others which have been even more difficult to model due to the multi-factorial nature of the condition and due to lack of discovery of a single causative gene such as age-related macular degeneration or Alzheimer’s syndrome. Thus a more relevant and accurate system will allow us to make better predictions on relevant therapeutic approaches.

The discovery of human pluripotent stem cells in 1998 followed by the technological advances to reprogram somatic cells to pluripotent-stem cell-like cells in 2006 has completely revolutionized the way we can now think about modelling human development and disease. This now coupled with genome editing technologies such as TALENS and CRISPRs have now set us up to develop in vitro models both 2D as well as 3D organoids, which can more precisely reflect the disease in the patients. These combinatorial technologies are already providing us with better tools and therapeutics in drug discovery or gene therapy.

This book summarizes both the technological advances in the field of generation of patient specific lines as well as various gene editing approaches followed by its applicability in various systems. The book will serve as a reference for the current state of the field as it:

-Provides a comprehensive overview of the status of the field of patients derived induced pluripotent stem cells.

-Describes the use of cardiac cells as a main featured component within the book.

-Examines drug toxicity analysis as a working example throughout the book.

chapter 1|22 pages

Human-Induced Pluripotent Stem Cells: Derivation

ByUthra Rajamani, Lindsay Lenaeus, Loren Ornelas, Dhruv Sareen

chapter 2|28 pages

Human-Induced Pluripotent Stem Cells: Banking and Characterization

ByUthra Rajamani, Lindsay Lenaeus, Loren Ornelas, Dhruv Sareen

chapter 3|36 pages

Genetic and Epigenetic Considerations in iPSC Technology

ByYoshiaki Tanaka, In-Hyun Park

chapter 4|14 pages

CRISPR-Based Genome Engineering in Human Stem Cells

ByThelma Garcia, Deepak A. Lamba

chapter 5|14 pages

Stem Cells for Parkinson’s Disease

ByDeepak A. Lamba

chapter 6|30 pages

Huntington’s Disease and Stem Cells

ByKaren Ring, Robert O’Brien, Ningzhe Zhang, Lisa M. Ellerby

chapter 8|16 pages

Role of iPSCs in Disease Modeling: Gaucher Disease and Related Disorders

ByDaniel K. Borger, Elma Aflaki, Ellen Sidransky

chapter 9|18 pages

Role of Induced Pluripotent Stem Cells in Urological Disease Modeling and Repair

ByMohammad Moad, Emma L. Curry, Craig N. Robson, Rakesh Heer

chapter 10|36 pages

Induced Pluripotent Stem Cells: A Research Tool and a Potential Therapy for RPE-Associated Blinding Eye Diseases

ByRuchi Sharma, Balendu Shekhar Jha, Kapil Bharti

chapter 11|22 pages

Modeling Neuroretinal Development and Disease in Stem Cells

ByDeepak A. Lamba