In vivo production of small interfering RNAs that mediate gene silencing

US9850487B2 · kind B2 · utility

Assignee

• University of Massachusetts · US

Inventors

• Phillip D. Zamore · Marlborough, US
• Juanita McLachlan · Worcester, US
• Gyorgy Hutvagner · Worcester, US
• Alla Grishok · Shrewsbury, US
• Craig C. Mello · Shrewsbury, US

Key dates

• Filing date: Sep 15, 2015
• Grant date: Dec 26, 2017
• Expiry date: Sep 15, 2035

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2830/008
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

The invention provides engineered RNA precursors that when expressed in a cell are processed by the cell to produce targeted small interfering RNAs (siRNAs) that selectively silence targeted genes (by cleaving specific mRNAs) using the cell's own RNA interference (RNAi) pathway. By introducing nucleic acid molecules that encode these engineered RNA precursors into cells in vivo with appropriate regulatory sequences, expression of the engineered RNA precursors can be selectively controlled both temporally and spatially, i.e., at particular times and/or in particular tissues, organs, or cells.