TY - JOUR
T1 - Advances in targeted delivery of small interfering RNA using simple bioconjugates
AU - Nielsen, Christoffer
AU - Kjems, Jørgen
AU - Sørensen, Kristine Rothaus
AU - Engelholm, Lars Henning
AU - Behrendt, Niels
PY - 2014/5
Y1 - 2014/5
N2 - INTRODUCTION: Development of drugs based on RNA interference by small interfering RNA (siRNA) has been progressing slowly due to a number of challenges associated with the in vivo behavior of siRNA. A central problem is controlling siRNA delivery to specific cell types. Here, we review existing literature on one type of strategy for solving the issue of cell-specific delivery of siRNA, namely delivering the siRNA as part of simple bioconjugate constructs.AREAS COVERED: This review presents current experience from strategies aimed at targeting siRNA to specific cell types, by associating the siRNA with a targeting moiety, in a simple bioconjugate construct. We discuss the use of different types of targeting moieties, as well as the different conjugation strategies employed for preparing these bioconjugate constructs that deliver the siRNA to target cells. We focus especially on the in-built or passive functionalities associated with each strategy, in order to identify key elements of successful siRNA delivery strategies with potential for further exploration.EXPERT OPINION: By evaluating the current literature on this subject, we identify strategies and concepts that are suitable for future studies, to enable the development of highly efficient simple bioconjugates for targeted siRNA delivery with therapeutic application.
AB - INTRODUCTION: Development of drugs based on RNA interference by small interfering RNA (siRNA) has been progressing slowly due to a number of challenges associated with the in vivo behavior of siRNA. A central problem is controlling siRNA delivery to specific cell types. Here, we review existing literature on one type of strategy for solving the issue of cell-specific delivery of siRNA, namely delivering the siRNA as part of simple bioconjugate constructs.AREAS COVERED: This review presents current experience from strategies aimed at targeting siRNA to specific cell types, by associating the siRNA with a targeting moiety, in a simple bioconjugate construct. We discuss the use of different types of targeting moieties, as well as the different conjugation strategies employed for preparing these bioconjugate constructs that deliver the siRNA to target cells. We focus especially on the in-built or passive functionalities associated with each strategy, in order to identify key elements of successful siRNA delivery strategies with potential for further exploration.EXPERT OPINION: By evaluating the current literature on this subject, we identify strategies and concepts that are suitable for future studies, to enable the development of highly efficient simple bioconjugates for targeted siRNA delivery with therapeutic application.
KW - Humans
KW - Molecular Targeted Therapy
KW - RNA Interference
KW - RNA, Small Interfering
U2 - 10.1517/17425247.2014.896898
DO - 10.1517/17425247.2014.896898
M3 - Review
C2 - 24669756
SN - 1742-5247
VL - 11
SP - 791
EP - 822
JO - Expert Opinion on Drug Delivery
JF - Expert Opinion on Drug Delivery
IS - 5
ER -