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US 11,826,434 B2Granted
Isolation of novel AAV's and uses thereof
AssigneeUniversity of Massachusetts
InventorsGuangping Gao, Phillip D. Zamore
Life of this patent
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Abstract
The invention in some aspects relates to isolated nucleic acids, compositions, and kits useful for identifying adeno-associated viruses in cells. In some aspects, the invention provides kits and methods for producing somatic transgenic animal models using recombinant AAV (rAAV) to an animal having at least one transgene that expresses a small interfering nucleic acid or at least one binding site for a miRNA.
Claims19
An isolated nucleic acid engineered to express a transgene, in a target tissue, in a subject,
- wherein the transgene is engineered to express an RNA transcript that comprises at least one binding site for a microRNA (miRNA) that is expressed in an off-target tissue of the subject, wherein the at least one miRNA binding site is positioned in the 5′ untranslated region (UTR) or 3′ UTR of the RNA transcript,
- wherein the transgene comprises a tissue-specific promoter selected from a thyroxin binding globulin (TBG) promoter, insulin promoter, glucagon promoter, somatostatin promoter, pancreatic polypeptide (PPY) promoter, synapsin-1 (Syn) promoter, creatine kinase (MCK) promoter, mammalian desmin (DES) promoter, .alpha.-myosin heavy chain (.alpha.-MHC) promoter, and a cardiac Troponin T (cTnT) promoter.
Dependencies are inferred from the claim text.