title: A genome editing primer for the hematologist.
abstract: Gene editing enables the site-specific modification of the genome. These technologies have rapidly advanced such that they have entered common use in experimental hematology to investigate genetic function. In addition, genome editing is becoming increasingly plausible as a treatment modality to rectify genetic blood disorders and improve cellular therapies. Genome modification typically ensues from site-specific double-strand breaks and may result in a myriad of outcomes. Even single-strand nicks and targeted biochemical modifications that do not permanently alter the DNA sequence (epigenome editing) may be powerful instruments. In this review, we examine the various technologies, describe their advantages and shortcomings for engendering useful genetic alterations, and consider future prospects for genome editing to impact hematology.
authors.affiliation: Division of Hematology/Oncology, Boston Children's Hospital, Boston, MA; Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, MA; and Department of Pediatrics, Harvard Medical School, Harvard Stem Cell Institute, Boston, MA.
authors.forename: Megan D
authors.identifier: 0000-0002-5011-9582
authors.initials: MD
authors.lastname: Hoban
authors.affiliation: Division of Hematology/Oncology, Boston Children's Hospital, Boston, MA; Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, MA; and Department of Pediatrics, Harvard Medical School, Harvard Stem Cell Institute, Boston, MA.
authors.forename: Daniel E
authors.identifier: 0000-0001-5076-7945
authors.initials: DE
authors.lastname: Bauer
mesh_terms: D000818:Animals; D000072669:Gene Editing / Q000379:methods*; D006402:Hematologic Diseases / Q000235:genetics* / Q000628:therapy*; D006801:Humans; D052416:Targeted Gene Repair / Q000379:methods*