Mitochondrial targeted RNA expression system and use thereof
AssigneeUniversity of Pittsburgh—Of the Commonwealth System of Higher Education
InventorsMichael J. Palladino, Alicia M. Palladino
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Abstract
Described herein is a mitochondrial-targeted RNA expression system (mtTRES) for delivery of RNA molecules to mitochondria. mtTRES vectors generate RNAs in vivo that are un-capped, non-polyadenylated, and actively directed to mitochondria. The disclosed vectors are capable of delivering either non-coding RNA molecules or RNA molecules encoding a protein of interest to the mitochondria. In particular, the disclosed vectors include (1) an RNAPIII initiation (promoter) sequence, (2) a non-coding leader sequence (NCL), (3) a mitochondrial translation initiation sequence and an ORF encoding a protein of interest, or a sequence encoding a non-coding RNA, and (4) an RNAPIII termination sequence.
Claims21
A vector comprising in the 5′ to 3′ direction:
- an RNA polymerase III (RNAPIII) promoter sequence;
- a non-coding mitochondrial leader sequence;
- a mitochondrial translation initiation sequence and an open reading frame (ORF) encoding a protein, or a sequence encoding a non-coding RNA molecule capable of inhibiting translation of a mitochondrial mRNA molecule;
- and
- an RNAPIII termination sequence,
- wherein the RNAPIII promoter sequence is a 5S rRNA RNAPIII promoter sequence at least 95% identical to SEQ ID NO:
- 9.
A vector comprising in the 5′ to 3′ direction:
- an RNA polymerase III (RNAPIII) promoter sequence;
- a non-coding mitochondrial leader sequence;
- a mitochondrial translation initiation sequence and an open reading frame (ORF) encoding a protein;
- and
- an RNAPIII termination sequence,
- wherein the RNAPIII promoter sequence is a 5S rRNA RNAPIII promoter sequence at least 95% identical to SEQ ID NO:
- 9.
A vector comprising in the 5′ to 3′ direction:
- an RNA polymerase III (RNAPIII) promoter sequence;
- a non-coding mitochondrial leader sequence;
- a mitochondrial translation initiation sequence and an open reading frame (ORF) encoding a protein, or a sequence encoding a non-coding RNA molecule capable of inhibiting translation of a mitochondrial mRNA molecule;
- and
- an RNAPIII termination sequence,
- wherein the 5S rRNA RNPAIII termination sequence is at least 95% identical to SEQ ID NO:
- 11.
Dependencies are inferred from the claim text.