Silencing of the Mutant Huntingtin Gene through CRISPR-Cas9 Improves the Mitochondrial Biomarkers in an In Vitro Model of Huntington’s Disease

Gary L. Dunbar, Sindhuja Koneru, Nivya Kolli, Michael Sandstrom, Panchanan Maiti, Julien Rossignol

Research output: Contribution to journalReview articlepeer-review

3 Scopus citations

Abstract

During the 25-year history of the American Society for Neural Therapy and Repair (ASNTR) there have been several breakthroughs in the area of neurotherapeutics, which was the case during the 2014–2105 year when one of us (GLD) had the privilege of serving as its president. During that year, the use of a newly developed gene-editing tool, the CRISPR-Cas9 system, started to skyrocket. Although scientists unraveled the use of “clustered regularly interspaced short palindromic repeats” (CRISPR) and its associated genes from the Cas family as an evolved mechanism of some bacterial and archaeal genomes to protect themselves from being hijacked by invasive viral genes, its use as a therapeutic tool was not fully appreciated until further research revealed how this system operated and how it might be developed technologically to manipulate genes of any species. By 2015, this technology had exploded to the point that close to 2,000 papers that used this technology were published during that year alone.

Original languageEnglish
Pages (from-to)460-463
Number of pages4
JournalCell Transplantation
Volume28
Issue number4
DOIs
StatePublished - Apr 1 2019

Keywords

  • gene therapy
  • neurodegeneration

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