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Modern Genome Editing Technologies in Huntington's Disease Research. / Malankhanova, Tuyana B.; Malakhova, Anastasia A.; Medvedev, Sergey P. et al.

In: Journal of Huntington's disease, Vol. 6, No. 1, 2017, p. 19-31.

Research output: Contribution to journal › Review article › peer-review

Harvard

Malankhanova, TB, Malakhova, AA, Medvedev, SP & Zakian, SM 2017, 'Modern Genome Editing Technologies in Huntington's Disease Research', Journal of Huntington's disease, vol. 6, no. 1, pp. 19-31. https://doi.org/10.3233/JHD-160222

APA

Malankhanova, T. B., Malakhova, A. A., Medvedev, S. P., & Zakian, S. M. (2017). Modern Genome Editing Technologies in Huntington's Disease Research. Journal of Huntington's disease, 6(1), 19-31. https://doi.org/10.3233/JHD-160222

Vancouver

Malankhanova TB, Malakhova AA, Medvedev SP, Zakian SM. Modern Genome Editing Technologies in Huntington's Disease Research. Journal of Huntington's disease. 2017;6(1):19-31. doi: 10.3233/JHD-160222

Author

Malankhanova, Tuyana B. ; Malakhova, Anastasia A. ; Medvedev, Sergey P. et al. / Modern Genome Editing Technologies in Huntington's Disease Research. In: Journal of Huntington's disease. 2017 ; Vol. 6, No. 1. pp. 19-31.

BibTeX

@article{39194520c0dd4461adfb5d265d67141a,
title = "Modern Genome Editing Technologies in Huntington's Disease Research",
abstract = "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.",
keywords = "disease modeling, Genome editing tools, Huntington's disease, induced pluripotent stem cells, isogenic cell lines",
author = "Malankhanova, {Tuyana B.} and Malakhova, {Anastasia A.} and Medvedev, {Sergey P.} and Zakian, {Suren M.}",
year = "2017",
doi = "10.3233/JHD-160222",
language = "English",
volume = "6",
pages = "19--31",
journal = "Journal of Huntington's disease",
issn = "1879-6397",
publisher = "IOS Press",
number = "1",

}

RIS

TY - JOUR

T1 - Modern Genome Editing Technologies in Huntington's Disease Research

AU - Malankhanova, Tuyana B.

AU - Malakhova, Anastasia A.

AU - Medvedev, Sergey P.

AU - Zakian, Suren M.

PY - 2017

Y1 - 2017

N2 - 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.

AB - 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.

KW - disease modeling

KW - Genome editing tools

KW - Huntington's disease

KW - induced pluripotent stem cells

KW - isogenic cell lines

UR - http://www.scopus.com/inward/record.url?scp=85016390139&partnerID=8YFLogxK

U2 - 10.3233/JHD-160222

DO - 10.3233/JHD-160222

M3 - Review article

C2 - 28128770

AN - SCOPUS:85016390139

VL - 6

SP - 19

EP - 31

JO - Journal of Huntington's disease

JF - Journal of Huntington's disease

SN - 1879-6397

IS - 1

ER -

ID: 8736111