Development of Therapeutic-Grade Small Interfering RNAs by Chemical Engineering

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Development of Therapeutic-Grade Small Interfering RNAs by Chemical Engineering. / Bramsen, Jesper B; Kjems, Jørgen.

In: Frontiers in Genetics, Vol. 3, 2012, p. 154.

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@article{99eb88c9379f43898073e76c0d9dd508,
title = "Development of Therapeutic-Grade Small Interfering RNAs by Chemical Engineering",
abstract = "Recent successes in clinical trials have provided important proof of concept that small interfering RNAs (siRNAs) indeed constitute a new promising class of therapeutics. Although great efforts are still needed to ensure efficient means of delivery in vivo, the siRNA molecule itself has been successfully engineered by chemical modification to meet initial challenges regarding specificity, stability, and immunogenicity. To date, a great wealth of siRNA architectures and types of chemical modification are available for promoting safe siRNA-mediated gene silencing in vivo and, consequently, the choice of design and modification types can be challenging to individual experimenters. Here we review the literature and devise how to improve siRNA performance by structural design and specific chemical modification to ensure potent and specific gene silencing without unwarranted side-effects and hereby complement the ongoing efforts to improve cell targeting and delivery by other carrier molecules.",
author = "Bramsen, {Jesper B} and J{\o}rgen Kjems",
year = "2012",
doi = "10.3389/fgene.2012.00154",
language = "English",
volume = "3",
pages = "154",
journal = "Frontiers in Genetics",
issn = "1664-8021",
publisher = "Frontiers Media S.A",

}

RIS

TY - JOUR

T1 - Development of Therapeutic-Grade Small Interfering RNAs by Chemical Engineering

AU - Bramsen, Jesper B

AU - Kjems, Jørgen

PY - 2012

Y1 - 2012

N2 - Recent successes in clinical trials have provided important proof of concept that small interfering RNAs (siRNAs) indeed constitute a new promising class of therapeutics. Although great efforts are still needed to ensure efficient means of delivery in vivo, the siRNA molecule itself has been successfully engineered by chemical modification to meet initial challenges regarding specificity, stability, and immunogenicity. To date, a great wealth of siRNA architectures and types of chemical modification are available for promoting safe siRNA-mediated gene silencing in vivo and, consequently, the choice of design and modification types can be challenging to individual experimenters. Here we review the literature and devise how to improve siRNA performance by structural design and specific chemical modification to ensure potent and specific gene silencing without unwarranted side-effects and hereby complement the ongoing efforts to improve cell targeting and delivery by other carrier molecules.

AB - Recent successes in clinical trials have provided important proof of concept that small interfering RNAs (siRNAs) indeed constitute a new promising class of therapeutics. Although great efforts are still needed to ensure efficient means of delivery in vivo, the siRNA molecule itself has been successfully engineered by chemical modification to meet initial challenges regarding specificity, stability, and immunogenicity. To date, a great wealth of siRNA architectures and types of chemical modification are available for promoting safe siRNA-mediated gene silencing in vivo and, consequently, the choice of design and modification types can be challenging to individual experimenters. Here we review the literature and devise how to improve siRNA performance by structural design and specific chemical modification to ensure potent and specific gene silencing without unwarranted side-effects and hereby complement the ongoing efforts to improve cell targeting and delivery by other carrier molecules.

U2 - 10.3389/fgene.2012.00154

DO - 10.3389/fgene.2012.00154

M3 - Journal article

VL - 3

SP - 154

JO - Frontiers in Genetics

JF - Frontiers in Genetics

SN - 1664-8021

ER -