Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery

摘要

A capsid-less AAV vector suitable for delivering exogenous DNA sequences into cells, tissues, organs, or a subject. The capsid-less AAV vector comprises two AAV inverted terminal repeats (ITRs) and an expression cassette, each of said ITRs having a hairpin structure and the cassette comprising at least one promoter operatively linked to an exogenous DNA sequence. The expression cassette is flanked on each end by one inverted terminal repeat and the vector does not encode AAV capsid proteins. Methods for producing and purifying capsid-less AAV vectors, and use of the same for therapeutic purposes are also provided.