ExLi4EvA, 2016. - 220 p. - This book offers insight into the modern tools of genome editing, their hurdles and their huge potential. A new era of in vivo genetic engineering has begun.
Site-specific endonucleases create double-strand breaks within the genome and can be targeted to literally any genetic mutation. Together with a repair template, a correction of the defective locus becomes possible.
Contents
Next-Generation Therapeutics: mRNA as a Novel Therapeutic Option for Single-Gene Disorders
Gene Delivery Technologies for Efficient Genome Editing: Applications in Gene Therapy
DNA Elements Tetris: A Strategy for Gene Correction
Gene Correction Technology and Its Impact on Viral Research and Therapy
Gene Editing in Adult Hematopoietic Stem Cells
Insulin Gene Therapy for Type 1 Diabetes Mellitus: Unique Challenges Require Innovative Solutions
Application of Genome Editing Technology to MicroRNA Research in Mammalians
Emerging Gene Correction Strategies for Muscular Dystrophies: Scientific Progress and Regulatory Impact
