Li, Lina (NHLBI)
Beley, Cyriaque (INSERM)
Voit, Thomas (INSERM)
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.
- The commercial applications of the technology relate to the field of gene therapy. It may offer significant advantages compared to existing methods of gene delivery and gene therapy.
- The AAV vectors described in the invention devoid the AAV capsid proteins and thus are not exposed to the adverse effects caused by immunogenicity.
- In contrast to the use of plasmid DNA for gene delivery, the AAV DNA of the invention seems to confer greater stability in cell nuclei, allowing prolonged expression compared to plasmid DNA.
- The vector DNA of the invention is not limited in size to the packageable size genome.
- The production of the AAV DNA vector is economical, simple and provides high yields.