scAAV.EF1a.miR26a.eGFP Plasmid

Case ID:

C10912

C10912: A plasmid to produce recombinant adeno-associated virus (AAV) that expresses microRNA-26a and green fluorescent protein

Value Proposition:

• miR-26a, cloned into the short intron that is part of the EF1a promoter unit, thus allowing simultaneous production of eGFP and miR-26a from a single transcript

• efficient expression of both miR-26a and eGFP

• equivalent amounts of mature miRNA & GFP as a controls

Technical Details:

Background

Therapeutic strategies based on modulation of microRNA (miRNA) activity hold great promise due to the ability of these small RNAs to potently influence cellular behavior. Although these therapeutic strategies are promising, delivery of RNA-based therapeutics to specific tissues for treating a variety of diseases has been problematic. Recently, gene therapy vectors based on adeno- associated virus (AAV) have been particularly promising as potential delivery systems. The development of self-complementary AAV (scAAV) vectors and the availability of AAV serotypes for improved transduction of specific target tissues has expanded the usefulness of this virus for therapeutic gene delivery. In particular, these advances allow highly efficient transduction of hepatocytes following systemic administration of scAAV8 vectors.

Technology

JHU researchers have developed scAAV.EF1a.miR26a.eGFP, a plasmid that can be used to produce recombinant adeno-associated virus (AAV) that expresses microRNA-26a and green fluorescent protein. Systemic administration of this miRNA in a mouse model of hepatocellular carcinoma (HCC) using AAV results in inhibition of cancer cell proliferation, induction of tumor-specific apoptosis, and dramatic protection from disease progression without toxicity. These findings suggest that delivery of miRNAs that are highly expressed and therefore tolerated in normal tissues but lost in disease cells may provide a general strategy for miRNA replacement therapies.

Looking for Partners:

The scAAV.EF1a.miR26a.eGFP plasmid can be used to produce recombinant adeno-associated virus (AAV) that expresses microRNA-26a and green fluorescent protein.

Publications/Associated Cases:

Kota J, Chivukula RR, O’Donnell KA, Wentzel EA, Montgomery CL, Hwang HW, Chang TC, Clark KR, Mendell JR, Mendell JT. (2009). Therapeutic microRNA delivery suppresses tumorigenesis in a murine liver cancer model. Cell 137(6):1005-1017.

Direct Link:

https://jhu.technologypublisher.com/technology/18740

Inventors:

Category(s):

Clinical and Disease Specializations, Clinical and Disease Specializations > Hepatology, Clinical and Disease Specializations > Oncology > Liver Cancer, Technology Classifications > Therapeutic Modalities > Gene Therapies, Technology Classifications > Therapeutic Modalities > Therapeutic Delivery Platforms, Clinical and Disease Specializations > Oncology, Technology Classifications > Therapeutic Modalities, Technology Classifications > Research Tools > Vectors & Plasmids,

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For Information, Contact:

Michael Woods

mwoods19@jh.edu

410-614-0300

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