title: RNA repair: a novel approach to gene therapy.
abstract: Treatment of genetic disorders by gene therapy has conventionally been attempted through the transfer of a wild type version of a gene to the cells of a patient harboring defective copies of a disease associated gene. Despite significant advances using this paradigm, several technical hurdles must still be overcome before this 'gene replacement' approach will become useful in the treatment of a variety of genetic maladies. Such limitations have led a number of researchers to begin to investigate alternative strategies to genetic therapy. Repair of mutant genetic instructions represents a fundamentally different approach to genetic therapy that may have significant advantages over gene replacement. Herein, we will discuss recent advances using repair of mutant RNAs as a novel means to correct genetic deficiencies.
authors.affiliation: Center for Genetic and Cellular Therapies, Departments of Surgery and Genetics, Duke University Medical Center, Durham, NC 27710, USA.
authors.forename: T
authors.initials: T
authors.lastname: Watanabe
authors.forename: B A
authors.initials: BA
authors.lastname: Sullenger
mesh_terms: D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D009154:Mutation; D016376:Oligonucleotides, Antisense / Q000235:genetics; D012313:RNA; D017393:RNA Editing; D022821:RNA Splice Sites / Q000235:genetics; D016337:RNA, Catalytic / Q000378:metabolism
chemical_list: D016376:Oligonucleotides, Antisense; D022821:RNA Splice Sites; D016337:RNA, Catalytic; D012313:RNA