TY - JOUR
T1 - Applications of RNA interference in the treatment of arthritis
AU - Rai, Muhammad Farooq
AU - Pan, Hua
AU - Yan, Huimin
AU - Sandell, Linda J.
AU - Pham, Christine T.N.
AU - Wickline, Samuel A.
N1 - Funding Information:
The work cited from Author's laboratories was partially supported by NIH grants R01AR067491 , R01HL073646 , R01DK102691 , R00 AR064837 , P30 AR073752 . The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health. All authors have read the journal's authorship agreement and approved the manuscript.
Publisher Copyright:
© 2019
PY - 2019/12
Y1 - 2019/12
N2 - RNA interference (RNAi) is a cellular mechanism for post-transcriptional gene regulation mediated by small interfering RNA (siRNA) and microRNA. siRNA-based therapy holds significant promise for the treatment of a wide-range of arthritic diseases. siRNA selectively suppresses the expression of a gene product and can thus achieve the specificity that is lacking in small molecule inhibitors. The potential use of siRNA-based therapy in arthritis, however, has not progressed to clinical trials despite ample evidence for efficacy in preclinical studies. One of the main challenges to clinical translation is the lack of a suitable delivery vehicle to efficiently and safely access diverse pathologies. Moreover, the ideal targets in treatment of arthritides remain elusive given the complexity and heterogeneity of these disease pathogeneses. Herein, we review recent preclinical studies that use RNAi-based drug delivery systems to mitigate inflammation in models of rheumatoid arthritis and osteoarthritis. We discuss a self-assembling peptide-based nanostructure that demonstrates the potential of overcoming many of the critical barriers preventing the translation of this technology to the clinic.
AB - RNA interference (RNAi) is a cellular mechanism for post-transcriptional gene regulation mediated by small interfering RNA (siRNA) and microRNA. siRNA-based therapy holds significant promise for the treatment of a wide-range of arthritic diseases. siRNA selectively suppresses the expression of a gene product and can thus achieve the specificity that is lacking in small molecule inhibitors. The potential use of siRNA-based therapy in arthritis, however, has not progressed to clinical trials despite ample evidence for efficacy in preclinical studies. One of the main challenges to clinical translation is the lack of a suitable delivery vehicle to efficiently and safely access diverse pathologies. Moreover, the ideal targets in treatment of arthritides remain elusive given the complexity and heterogeneity of these disease pathogeneses. Herein, we review recent preclinical studies that use RNAi-based drug delivery systems to mitigate inflammation in models of rheumatoid arthritis and osteoarthritis. We discuss a self-assembling peptide-based nanostructure that demonstrates the potential of overcoming many of the critical barriers preventing the translation of this technology to the clinic.
UR - http://www.scopus.com/inward/record.url?scp=85069924790&partnerID=8YFLogxK
U2 - 10.1016/j.trsl.2019.07.002
DO - 10.1016/j.trsl.2019.07.002
M3 - Review article
C2 - 31351032
AN - SCOPUS:85069924790
SN - 1931-5244
VL - 214
SP - 1
EP - 16
JO - Translational Research
JF - Translational Research
ER -