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Silencing disease genes in the laboratory and the clinic

Silencing disease genes in the laboratory and the clinic
Silencing disease genes in the laboratory and the clinic
Synthetic nucleic acids are commonly used laboratory tools for modulating gene expression and have the potential to be widely used in the clinic. Progress towards nucleic acid drugs, however, has been slow and many challenges remain to be overcome before their full impact on patient care can be understood. Antisense oligonucleotides (ASOs) and small interfering RNAs (siRNAs) are the two most widely used strategies for silencing gene expression. We first describe these two approaches and contrast their relative strengths and weaknesses for laboratory applications. We then review the choices faced during development of clinical candidates and the current state of clinical trials. Attitudes towards clinical development of nucleic acid silencing strategies have repeatedly swung from optimism to depression during the past 20 years. Our goal is to provide the information needed to design robust studies with oligonucleotides, making use of the strengths of each oligonucleotide technology.
antisense oligonucleotides, siRNA, mRNA, gene silencing, therapeutics
365-379
Watts, Jonathan K.
c4de85ee-aaa3-4e7d-99b3-147a4de4f01c
Corey, David R.
ce328d93-1c07-4ab0-952c-da300f6738f9
Watts, Jonathan K.
c4de85ee-aaa3-4e7d-99b3-147a4de4f01c
Corey, David R.
ce328d93-1c07-4ab0-952c-da300f6738f9

Watts, Jonathan K. and Corey, David R. (2012) Silencing disease genes in the laboratory and the clinic. [in special issue: The Cell Biology of Disease] The Journal of Pathology, 226 (2), 365-379. (doi:10.1002/path.2993). (PMID:22069063)

Record type: Article

Abstract

Synthetic nucleic acids are commonly used laboratory tools for modulating gene expression and have the potential to be widely used in the clinic. Progress towards nucleic acid drugs, however, has been slow and many challenges remain to be overcome before their full impact on patient care can be understood. Antisense oligonucleotides (ASOs) and small interfering RNAs (siRNAs) are the two most widely used strategies for silencing gene expression. We first describe these two approaches and contrast their relative strengths and weaknesses for laboratory applications. We then review the choices faced during development of clinical candidates and the current state of clinical trials. Attitudes towards clinical development of nucleic acid silencing strategies have repeatedly swung from optimism to depression during the past 20 years. Our goal is to provide the information needed to design robust studies with oligonucleotides, making use of the strengths of each oligonucleotide technology.

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e-pub ahead of print date: 9 November 2011
Published date: January 2012
Keywords: antisense oligonucleotides, siRNA, mRNA, gene silencing, therapeutics
Organisations: Chemistry

Identifiers

Local EPrints ID: 340221
URI: http://eprints.soton.ac.uk/id/eprint/340221
PURE UUID: 7786da71-feea-4542-8e2d-d0c5efcc749c

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Date deposited: 15 Jun 2012 12:31
Last modified: 16 Dec 2019 20:38

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Contributors

Author: Jonathan K. Watts
Author: David R. Corey

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