Directed gene modification via triple helix formation.
The ability to selectively target mammalian genes and disrupt or restore their function would represent an important advance in gene therapy. Mutation of a single nucleotide can often result in a non-functional gene product. Reversion of defective genes to their correct sequences could lead to permanent cures for patients with many genetic diseases. Molecules such as triplex forming oligonucleotides (TFOs) and peptide nucleic acids (PNAs) are currently being employed to bind to double-stranded DNA. Efficient targeting of genomic DNA with these molecules will be the initial step in gene modification.
Gorman
L
L
Department of Therapeutic Radiology, Yale University School of Medicine, Boyer Center for Molecular Medicine, New Haven, CT 06536, USA.
Glazer
P M
PM
D000818:Animals; D001483:Base Sequence; D004247:DNA / Q000737:chemistry* / Q000235:genetics*; D018390:Gene Targeting; D030342:Genetic Diseases, Inborn / Q000235:genetics / Q000628:therapy; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D051379:Mice; D016297:Mutagenesis, Site-Directed; D009690:Nucleic Acid Conformation; D009838:Oligodeoxyribonucleotides / Q000737:chemistry / Q000235:genetics; D020135:Peptide Nucleic Acids / Q000737:chemistry / Q000235:genetics; D011995:Recombination, Genetic
D009838:Oligodeoxyribonucleotides; D020135:Peptide Nucleic Acids; D004247:DNA