title: Genome Editing of the Blood: Opportunities and Challenges.
abstract: The ability to remove blood cells, including hematopoietic stem cells (HSCs), from a person and then re-transplant them (hematopoietic stem cell transplantation (HSCT) is a well-established treatment paradigm that can be used in both the autologous setting or in the allogeneic setting. Using allogeneic HSCT can cure different genetic diseases of the blood but has significant limitations. An alternative to allogeneic HSCT is to transplant genetically modified HSCs instead. A powerful approach to the precision modification of HSCs is to use genome editing whereby the genome is modified with spatial precision (at an exact location) in the genome and sometimes with nucleotide precision (the exact nucleotide changes are introduced). The progress and challenges of genome editing of blood are discussed.
authors.affiliation: Dept. of Pediatrics MC5462, Stanford University, Stanford, CA 94305, mporteus@stanford.edu.
authors.forename: Matthew H
authors.initials: MH
authors.lastname: Porteus
keywords: CRISPR/Cas9; Genome editing; TAL effector nuclease; engineered nuclease; homologous recombination; non-homologous end-joining; zinc finger nuclease