Development of Human Genome Editing Tools for the Study of Genetic Variations and Gene Therapies
Contributors: Church, George M.; Seidman, Jonathan; Weiss, Ron; Rossi, Derrick; Rinn, John
Published: 2013
Language: eng
Description: The human genome encodes information that instructs human development, physiology, medicine, and evolution. Massive amount of genomic data has generated an ever-growing pool of hypothesis. Genome editing, broadly defined as targeted changes to the genome, posits to deliver the promise of genomic revolution to transform basic science and personalized medicine. This thesis aims to contribute to this scientific endeavor with a particular focus on the development of effective human genome engineering tools.
Notes: Keywords: CRISPR/cas, gene therapy, human genome engineering, TALE, targeted nucleases.

Thesis Ph.D. Harvard University 2013