Abstract: An isolated 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 for producing and purifying this nucleic acid molecule, and use of the same for therapeutic purposes are also provided.
March 12, 2012
Date of Patent:
March 21, 2017
ASSOCIATION INSTITUT DE MYOLOGIE; UNIVERSITÉ PIERRE ET MARIE CURIE (PARIS 6); CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE; INSTITUT NATIONAL DE LA SANTÉ DE LAD RECHERCHE MÉDICALE, THE UNITED STATES OF AMERICA, AS REPRESENTED BY THE DEPARTMENT OF HEALTH AND HUMAN SERVICES, NATIONAL INSTITUTES OF HEALTH
Luis Garcia, Cyriaque Beley, Thomas Voit, Robert Michael Kotin, Lina Li