Capsid-Free AAV Vectors for Gene Delivery and Their Use for Gene Therapy
The invention concerns novel capsid-free AAV vectors that can be used for gene delivery and gene therapy applications. The invention provides for a linear nucleic acid molecule comprising in this order: a first adeno-associated virus (AAV) inverted terminal repeat (ITR), a nucleotide sequence of interest, and a second AAV ITR, wherein said nucleic acid molecule is devoid of AAV capsid protein coding sequences. The said nucleic acid molecule can be applied to a host at repetition without eliciting an immune response. Methods of producing and purifying this nucleic acid molecule, as well as its use for gene transfer and gene therapy are also described.
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Inventors:
Robert Kotin (NHLBI) ➽ more inventions...
Lina Li (NHLBI) ➽ more inventions...
Luis Garcia
Cyriaque Beley
Thomas Voit
Intellectual Property:
U.S. Pat: 9,598,703 issued 2017-03-21
PCT Application No. PCT/EP2012/054303
US Application No. 61/452,071
and various international counterparts
Publications:
Li L, et al. PMID 23936358
Licensing Contact:
John Devany,
Email: john.devany@nih.gov
Phone: 301-594-4090
OTT Reference No: E-241-2010-0
Updated: Oct 12, 2016