Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery
AssigneesAssociation 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
InventorsLuis Garcia, Cyriaque Beley, Thomas Voit, Robert Michael Kotin, Lina Li
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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.
Claims17
An isolated linear capsid-free 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.
A method for delivering a nucleotide sequence of interest mediating the expression of exogenous DNA in a cell, an organ or tissue of a subject, comprising administering to said subject an isolated linear capsid-free nucleic acid molecule of 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.
A method for mediating the expression of an exogenous DNA in a cell, an organ or tissue of a subject, comprising administering to said subject an isolated linear capsid-free 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.
Dependencies are inferred from the claim text.