COMPOSITIONS AND METHODS FOR THE PRODUCTION OF scAAV
AssigneeVoyager Therapeutics, Inc.
InventorsJames McLaughlin, Robert Kotin
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Abstract
The present invention is directed to viral vectors and methods of their production and use.
Claims73
A parvoviral ITR nucleotide sequence comprising an engineered Rep binding sequence region (eRBSR) wherein the parvoviral ITR nucleotide sequence is capable of forming a double-stranded Rep binding sequence region.
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An oligonucleotide comprising an engineered Rep binding sequence region (eRBSR), wherein the eRBSR is from about 4 nucleotides to about 64 nucleotides in length.
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A polynucleotide comprising, in the 5′ to 3′ direction, a first parvoviral inverted terminal repeat sequence, a first heterologous sequence, a parvoviral inverted terminal repeat (ITR) nucleotide sequence comprising an engineered Rep binding sequence region, a second heterologous sequence, wherein the second heterologous sequence is complementary to the first heterologous sequence, and a second parvoviral inverted terminal repeat sequence.
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A method of producing a self-complementary adeno-associated viral particle comprising
- a. introducing into competent bacterial cells
- i. a payload construct vector comprising a heterologous sequence flanked on each side by a parvoviral ITR sequence, wherein either of the parvoviral ITR sequences comprises an eRBSR, to produce a payload construct expression vector;
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- ii. one or more viral construct vector(s) comprising parvoviral rep and/or cap gene sequences under the control of one or more regulatory elements to produce a viral construct expression vector;
- b. introducing into viral replication cells
- i. the payload construct expression vector produced in step (a.i) to produce a payload construct particle;
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- ii. the viral construct expression vector(s) produced in step (a.ii) to produce a viral construct particle;
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- c. co-infecting a viral replication cell with the payload construct viral particle produced in step (b.i) and the one or more viral construct viral particle(s) of step (b.ii) to produce a self-complementary adeno-associated viral (scAAV) particle.
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Dependencies are inferred from the claim text.