title: Development of nuclease-mediated site-specific genome modification.
abstract: Genome engineering is an emerging strategy to treat monogenic diseases that relies on the use of engineered nucleases to correct mutations at the nucleotide level. Zinc finger nucleases can be designed to stimulate homologous recombination-mediated gene targeting at a variety of loci, including genes known to cause the primary immunodeficiencies (PIDs). Recently, these nucleases have been used to correct disease-causing mutations in human cells, as well as to create new animal models for human disease. Although a number of hurdles remain before they can be used clinically, engineered nucleases hold increasing promise as a therapeutic tool, particularly for the PIDs.
authors.affiliation: Department of Pediatrics, Stanford University School of Medicine, Stanford, CA 94305, USA.
authors.forename: Stacey E
authors.initials: SE
authors.lastname: Wirt
authors.forename: Matthew H
authors.initials: MH
authors.lastname: Porteus
mesh_terms: D000818:Animals; D030801:Animals, Genetically Modified; D002478:Cells, Cultured; D004262:DNA Restriction Enzymes / Q000235:genetics / Q000378:metabolism / Q000502:physiology*; D018390:Gene Targeting / Q000379:methods* / Q000639:trends; D005818:Genetic Engineering / Q000379:methods* / Q000639:trends; D015894:Genome, Human; D006801:Humans; D016335:Zinc Fingers / Q000235:genetics
chemical_list: D004262:DNA Restriction Enzymes