TY - JOUR
T1 - Chemical modification of small interfering RNA
AU - Bramsen, Jesper Bertram
AU - Kjems, Jørgen
PY - 2011
Y1 - 2011
N2 - Chemically synthesized siRNAs are widely used for gene silencing. For in vitro applications, stability, delivery, and immunological issues are rarely problematic, but for in vivo applications the situation is different. Limited stability, undesirable pharmacokinetic behaviour, and unanticipated side effects from the immune system call for more careful structural siRNA design and inclusion of chemical modifications at selected positions. Also the notion that siRNA induces significant off-target silencing of many non-related genes has promted new effective measures to enhance specificity. The scope of this review is to provide a simple guide to successful chemical and structural modification of siRNAs with improved activity, stability, specificity, and low toxicity.
AB - Chemically synthesized siRNAs are widely used for gene silencing. For in vitro applications, stability, delivery, and immunological issues are rarely problematic, but for in vivo applications the situation is different. Limited stability, undesirable pharmacokinetic behaviour, and unanticipated side effects from the immune system call for more careful structural siRNA design and inclusion of chemical modifications at selected positions. Also the notion that siRNA induces significant off-target silencing of many non-related genes has promted new effective measures to enhance specificity. The scope of this review is to provide a simple guide to successful chemical and structural modification of siRNAs with improved activity, stability, specificity, and low toxicity.
KW - Animals
KW - Drug Design
KW - Humans
KW - RNA, Small Interfering
KW - Ribonucleases
U2 - 10.1007/978-1-61779-037-9_5
DO - 10.1007/978-1-61779-037-9_5
M3 - Journal article
C2 - 21431680
SN - 1064-3745
VL - 721
SP - 77
EP - 103
JO - Methods in Molecular Biology
JF - Methods in Molecular Biology
IS - 1
ER -