Targeted gene repair and its application to neurodegenerative disorders

Hetal Parekh-Olmedo, Dimitri Krainc, Eric B. Kmiec*

*Corresponding author for this work

Research output: Contribution to journalReview article

10 Scopus citations

Abstract

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.

Original languageEnglish (US)
Pages (from-to)495-498
Number of pages4
JournalNeuron
Volume33
Issue number4
DOIs
Publication statusPublished - Feb 14 2002

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ASJC Scopus subject areas

  • Neuroscience(all)

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