Delivery of therapeutic RNAs via ARRDC1-mediated microvesicles
AssigneePresident and Fellows of Harvard College
InventorQuan Lu
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Abstract
Methods, systems, compositions and strategies for the delivery of RNA into cells in vivo, ex vivo, or in vitro via ARMMs are provided. In some aspects, ARMMs containing fusion proteins of ARRDC1 fused to an RNA binding protein or an RNA binding protein fused to a WW domain are provided. In some aspects, ARMMs containing binding RNAs associated with cargo RNAs are provided. In other aspects, cargo RNAs associated with a binding RNA, such as a TAR element, are loaded into ARMMs via ARRDC1 fusion proteins containing an RNA binding protein, such as trans-activator of transcription (Tat) protein.
Claims28
An arrestin domain-containing protein 1 (ARRDC1)-mediated microvesicle (ARMM) comprising:
- a lipid bilayer,
- an ARRDC1 protein or an ARRDC1-protein variant, wherein the ARRDC1-protein variant comprises an amino acid sequence that is at least about 95% identical to the amino acid sequence of any one of SEQ ID NOs:
- 15-17,
- an RNA binding protein, wherein the ARRDC1 protein or the ARRDC1-protein variant is associated with the RNA binding protein, and
- a binding RNA, wherein the binding RNA is not a guide RNA, and further wherein the binding RNA is associated with the RNA binding protein.
An ARRDC1 fusion protein comprising:
- an ARRDC1 protein or an ARRDC1-protein variant, wherein the ARRDC1 variant comprises an amino acid sequence that is at least about 95% identical to amino acid sequence of any one of SEQ ID NOs:
- 15-17, and
- an RNA binding protein,
- wherein the RNA binding protein is associated with a binding RNA, and further wherein the binding RNA is not a guide RNA.
Dependencies are inferred from the claim text.