Modern Genome Editing Technologies in Huntington's Disease Research

Tuyana B. Malankhanova, Anastasia A. Malakhova, Sergey P. Medvedev, Suren M. Zakian

Research output: Contribution to journalReview articlepeer-review

15 Citations (Scopus)


The development of new revolutionary technologies for directed gene editing has made it possible to thoroughly model and study NgAgo human diseases at the cellular and molecular levels. Gene editing tools like ZFN, TALEN, CRISPR-based systems, NgAgo and SGN can introduce different modifications. In gene sequences and regulate gene expression in different types of cells including induced pluripotent stem cells (iPSCs). These tools can be successfully used for Huntington's disease (HD) modeling, for example, to generate isogenic cell lines bearing different numbers of CAG repeats or to correct the mutation causing the disease. This review presents common genome editing technologies and summarizes the progress made in using them in HD and other hereditary diseases. Furthermore, we will discuss prospects and limitations of genome editing in understanding HD pathology.

Original languageEnglish
Pages (from-to)19-31
Number of pages13
JournalJournal of Huntington's disease
Issue number1
Publication statusPublished - 2017


  • disease modeling
  • Genome editing tools
  • Huntington's disease
  • induced pluripotent stem cells
  • isogenic cell lines


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