CRISPR-Cas9 Mediated Gene-Silencing of the Mutant Huntingtin Gene in an In Vitro Model of Huntington’s Disease

@inproceedings{Kolli2017CRISPRCas9MG,
  title={CRISPR-Cas9 Mediated Gene-Silencing of the Mutant Huntingtin Gene in an In Vitro Model of Huntington’s Disease},
  author={Nivya Kolli and Ming Lu and Panchanan Maiti and Julien Rossignol and Gary L Dunbar},
  booktitle={International journal of molecular sciences},
  year={2017}
}
Huntington's disease (HD) is a fatal neurodegenerative genetic disease characterized by a loss of neurons in the striatum. It is caused by a mutation in the Huntingtin gene (HTT) that codes for the protein huntingtin (HTT). The mutant Huntingtin gene (mHTT) contains extra poly-glutamine (CAG) repeats from which the translated mutant huntingtin proteins… CONTINUE READING