Targeted gene repair and its application to neurodegenerative disorders.
Synthetic DNA oligonucleotides can direct the exchange of single nucleotides within coding regions of mammalian genes by hybridizing to their complementary sequence in the chromosome and creating a recombination joint structure with a single mismatched base pair. Inherent DNA repair processes recognize the mismatch and resolve it using the DNA sequence of the oligonucleotide vector as the template. This gene surgery approach can be used to repair mutations or to disrupt tri-nucleotide repeats in dysfunctional genes responsible for neurological disorders.
Parekh-Olmedo
Hetal
H
Department of Biological Sciences, Delaware Biotechnology Institute, University of Delaware, Newark, DE 19716, USA.
Krainc
Dimitri
D
Kmiec
Eric B
EB
D000818:Animals; D004247:DNA / Q000235:genetics / Q000627:therapeutic use; D018390:Gene Targeting / Q000379:methods* / Q000639:trends*; D005822:Genetic Vectors / Q000627:therapeutic use*; D006801:Humans; D009154:Mutation / Q000235:genetics*; D019636:Neurodegenerative Diseases / Q000235:genetics / Q000503:physiopathology / Q000628:therapy*; D015345:Oligonucleotide Probes / Q000627:therapeutic use*; D012313:RNA / Q000235:genetics / Q000627:therapeutic use
D015345:Oligonucleotide Probes; D012313:RNA; D004247:DNA