Novel short hairpin RNA compositions, methods of making and applications thereof

摘要

Provided herein are novel synthetic short hairpin RNA (shRNA) molecules and compositions and kits comprising such molecules, as well as methods of making and using these molecules, compositions, and kits.

主权项

1. A synthetic short hairpin RNA (shRNA) molecule designed to silence the expression of a target gene, the synthetic shRNA molecule comprising:
(a) a 5′ arm and a 3′ arm comprising:
(i) a stem region comprising 16, 17 or 18 base pairs, the base pairs comprising nucleotides from the 5′ arm paired with nucleotides from the 3′ arm, and(ii) one or more unpaired nucleotides at the 5′ terminal end of the 5′ arm and one or more unpaired nucleotides at the 3′ terminal end of the 3′ arm; and b) a loop region comprising 4 nucleotides that connects the 5′ arm to the 3′ arm, wherein the synthetic shRNA molecule is processed by Argonaute 2 (Ago2) in a Dicer-independent manner.