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Tri-AAV vector to deliver full-length dystrophin gene expression cassette

IP Title: HYBRID-AAV VECTORS TO DELIVER LARGE GENE EXPRESSION CASSETTE

Detailed Technology Description: None

Application Date: May 28, 2009

Application No.: 8,236,557

Others

*Abstract: Mutations in the dystrophin gene result in Duchenne muscular dystrophy (DMD), Becker muscular dystrophy (BMD) and X-linked dilated cardiomyopathy (XLDC). There is no cure for these devastating diseases. Adeno- associated virus (AAV) is the only gene therapy vector that can efficiently deliver a therapeutic gene to all body muscles. Therefore, replacing the defective dystrophin gene with AAV gene therapy holds great therapeutic promise. To date, AAV vectors have only been shown to express truncated versions of the dystrophin gene but not full-length protein. The present invention describes a novel tri-AAV system to efficiently deliver the full-length dystrophin coding sequence. This strategy will allow efficient expression of the full-length dystrophin protein from AAV vectors. This invention will lead to a breakthrough in AAV-mediated DMD gene therapy. The strategies described in this invention can also be applied to deliver other large genes for diagnostic and/or therapeutic purpose.POTENTIAL AREAS OF APPLICATIONS:o Expansion of AAV packaging capacity to 15kb to meet the need of DMD, BMD and XLDC gene therapyo Usage in expressing large transcriptional regulatory elements to fine tune gene expression from any AAV vectoro Enhanced specificity and efficiency of any AAV gene therapyMAIN ADVANTAGES OF INVENTION:o Expression of a full-length protein has significant therapeutic advantage over the current truncated proteins in restoring the biological function of various genesSTATE OF DEVELOPMENT:o PreclinicalPATENT STATUS:o Issued 8,236,557INVENTOR(S):o D. DuanCONTACT INFO:Paul Hippenmeyer, Sr. Licensing & Business Development AssociateOffice of Technology Management and Industry RelationsEmail: hippenmeyerp@missouri.eduPhone: 573-882-0470

*IP Issue Date: Aug 7, 2012

*IP Publication Date: Jan 7, 2010

*Principal Investigator: Name: Dongsheng Duan, Professor, Molecular Microbiology & Immunology
Department:

Name: Arka Ghosh, Post Doc Fellow
Department:

Name: Yongping Yue, Research Specialist Lead
Department:

Country/Region: USA

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