RNA interference-based gene therapy for successful treatment of rheumatoid arthritis.
BACKGROUND
RNA interference (RNAi) is a powerful endogenous process initiated by short double-stranded RNAs, which results in sequence-specific posttranscriptional gene silencing. The possibility of blocking the expression of any protein carries huge expectations for potential therapeutic applications in a wide range of diseases. For clinical development, however, the use of RNAi-based therapeutics has to overcome major obstacles, mainly targeted delivery and safety issues.


OBJECTIVE
In this short review, we provide an overview of specifications for RNAi-based gene therapy in rheumatoid arthritis (RA) and discuss recent progresses in the development of efficient silencing, focusing on expression of short hairpin RNAs.


CONCLUSIONS
Combining advances in RNAi methodology with gene therapy technology opens avenues for rapid applications to RA.
Courties
Gabriel
G
Inserm, U844, Université Montpellier 1, CHU Lapeyronie, Montpellier, France.
Presumey
Jessy
J
Duroux-Richard
Isabelle
I
Jorgensen
Christian
C
Apparailly
Florence
F
D000818:Animals; D001172:Arthritis, Rheumatoid / Q000235:genetics* / Q000628:therapy*; D015316:Genetic Therapy / Q000379:methods* / Q000639:trends; D006801:Humans; D034622:RNA Interference / Q000502:physiology*; D034741:RNA, Small Interfering / Q000627:therapeutic use*
D034741:RNA, Small Interfering