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Zinc finger nuclease technology: advances and obstacles in modelling and treating genetic disorders

Zinc finger nuclease technology: advances and obstacles in modelling and treating genetic disorders
Zinc finger nuclease technology: advances and obstacles in modelling and treating genetic disorders
Zinc finger nucleases (ZFNs) are engineered restriction enzymes designed to target specific DNA sequences within the genome. Assembly of zinc finger DNA-binding domain to a DNA-cleavage domain enables the enzyme machinery to target unique locus in the genome and invoke endogenous DNA repair mechanisms. This machinery offers a versatile approach in allele editing and gene therapy. Here we discuss the architecture of ZFNs and strategies for generating targeted modifications within the genome. We review advances in gene therapy and modelling of the disease using these enzymes and finally, discuss the practical obstacles in using this technology.
0378-1119
1-5
Jabalameli, H.Reza
55ae0f38-c364-410d-b6ac-c582ac8a7eea
Zahednasab, Hamid
467cdf50-ec29-4ca8-8687-971764ae2654
Karimi-Moghaddam, Amin
d5b0a7f9-9380-44fe-9616-e03efcc67e4e
Jabalameli, M.Reza
d533e702-7a6b-4f2d-8947-352ea1dd769b
Jabalameli, H.Reza
55ae0f38-c364-410d-b6ac-c582ac8a7eea
Zahednasab, Hamid
467cdf50-ec29-4ca8-8687-971764ae2654
Karimi-Moghaddam, Amin
d5b0a7f9-9380-44fe-9616-e03efcc67e4e
Jabalameli, M.Reza
d533e702-7a6b-4f2d-8947-352ea1dd769b

Jabalameli, H.Reza, Zahednasab, Hamid, Karimi-Moghaddam, Amin and Jabalameli, M.Reza (2015) Zinc finger nuclease technology: advances and obstacles in modelling and treating genetic disorders. Gene, 558 (1), 1-5. (doi:10.1016/j.gene.2014.12.044). (PMID:25536166)

Record type: Article

Abstract

Zinc finger nucleases (ZFNs) are engineered restriction enzymes designed to target specific DNA sequences within the genome. Assembly of zinc finger DNA-binding domain to a DNA-cleavage domain enables the enzyme machinery to target unique locus in the genome and invoke endogenous DNA repair mechanisms. This machinery offers a versatile approach in allele editing and gene therapy. Here we discuss the architecture of ZFNs and strategies for generating targeted modifications within the genome. We review advances in gene therapy and modelling of the disease using these enzymes and finally, discuss the practical obstacles in using this technology.

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Accepted/In Press date: 19 December 2014
e-pub ahead of print date: 20 December 2014
Published date: 1 March 2015
Organisations: Human Development & Health

Identifiers

Local EPrints ID: 396188
URI: http://eprints.soton.ac.uk/id/eprint/396188
ISSN: 0378-1119
PURE UUID: bea58a87-ac8a-483a-8eac-c927eb05f354
ORCID for M.Reza Jabalameli: ORCID iD orcid.org/0000-0002-7762-0529

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Date deposited: 07 Jun 2016 11:01
Last modified: 15 Mar 2024 00:47

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Contributors

Author: H.Reza Jabalameli
Author: Hamid Zahednasab
Author: Amin Karimi-Moghaddam
Author: M.Reza Jabalameli ORCID iD

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