AAV virions with decreased immunoreactivity and uses therefor
AssigneeGenzyme Corporation
InventorsAlejandra Arbetman, Peter Colosi, Michael Lochrie, Richard Surosky
Life of this patent
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Abstract
Methods of making and using recombinant AAV virions with decreased immunoreactivity are described. The recombinant AAV virions include mutated capsid proteins or are derived from non-primate mammalian AAV serotypes and isolates that display decreased immunoreactivity relative to AAV-2.
Claims43
A mutated adeno-associated virus (AAV) capsid protein that when present in an AAV virion imparts decreased immunoreactivity to the virion as compared to the corresponding wild-type virion.
A method of delivering a recombinant AAV virion to a cell or tissue of a vertebrate subject, said method comprising:
- (a) providing a recombinant AAV virion, wherein said AAV virion comprises
- (i) a non-primate, mammalian adeno-associated virus (AAV) capsid protein that when present in an AAV virion imparts decreased immunoreactivity to the virion as compared to immunoreactivity of primate AAV-2;
- and
- (ii) a heterologous nucleic acid molecule encoding a therapeutic protein operably linked to control elements capable of directing the in vivo transcription and translation of said protein;
- (b) delivering said recombinant AAV virion to said cell or tissue, whereby said protein is expressed at a level that provides a therapeutic effect.
Dependencies are inferred from the claim text.