DELIVERY METHODS AND COMPOSITIONS FOR NUCLEASE-MEDIATED GENOME ENGINEERING
The present disclosure is in the field of genome engineering, particularly targeted modification of the genome of a cell.
1 .- 11 . (canceled)
12 . A method of introducing a nucleic acid into a cell of a subject, the method comprising:
administering to the subject at least one adeno-associated vector (AAV) comprising a donor molecule and at least one steroid and/or at least one B-cell inhibitor.
13 . The method of claim 12 , wherein the donor molecule comprising a sequence encoding a transgene.
14 . The method of claim 12 , wherein the subject is a mammal and the transgene encodes a therapeutic protein, and further wherein the mammal becomes tolerized to the therapeutic protein.
15 . The method of claim 12 , wherein the subject has a hemophilia and the donor molecule encodes a clotting factor.
16 . The method of claim 15 , wherein the clotting factor is Factor VIII or Factor IX.
17 .- 18 . (canceled)
19 . The method of claim 12 , wherein the administering comprises administering at least one steroid.
20 . The method of claim 19 , wherein the steroid is methylprednisolone or prednisolone.
21 . The method of claim 12 , wherein the administering comprises administering at least one B-cell inhibitor.
22 . The method of claim 21 , wherein the B-cell inhibitor is rituximab.
23 . The method of claim 12 , wherein the at least one steroid and/or at least one B-cell inhibitor is administered after the donor molecule.
24 . The method of claim 12 , wherein the at least one steroid and/or at least one B-cell inhibitor is administered simultaneously with the donor molecule.
25 . The method of claim 12 , wherein the at least one steroid and/or at least one B-cell inhibitor is administered before the donor molecule.
26 . The method of claim 12 , wherein the at least one steroid and the at least one B-cell inhibitor is administered.