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

被引:5
|
作者
Dunbar, Gary L. [1 ,2 ,3 ,4 ]
Koneru, Sindhuja [1 ,2 ]
Kolli, Nivya [1 ,2 ]
Sandstrom, Michael [2 ,3 ]
Maiti, Panchanan [1 ,2 ,4 ,5 ]
Rossignol, Julien [1 ,2 ,6 ]
机构
[1] Cent Michigan Univ, Field Neurosci Inst, Lab Restorat Neurol, Mt Pleasant, MI 48859 USA
[2] Cent Michigan Univ, Program Neurosci, Mt Pleasant, MI 48859 USA
[3] Cent Michigan Univ, Dept Psychol, Mt Pleasant, MI 48859 USA
[4] Saginaw Valley State Univ, Dept Biol, Saginaw, MI USA
[5] St Marys Michigan, Field Neurosci Inst, Saginaw, MI USA
[6] Cent Michigan Univ, Coll Med, Mt Pleasant, MI 48859 USA
关键词
gene therapy; neurodegeneration; REPEATS; RNA;
D O I
10.1177/0963689719840662
中图分类号
Q813 [细胞工程];
学科分类号
摘要
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.
引用
收藏
页码:460 / 463
页数:4
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