Materials And Methods For Treatment Of Disorders Associated With The IGHMBP2 Gene

Abstract

The disclosure provides gene therapy vectors, such as adeno-associated virus (AAV), designed for treatment of an immunoglobulin- μ binding protein 2 (IGHMB P2)-related disorder. 20 26 22 63 76 01 S ep 2 02 6 2 0 2 6 2 2 6 3 7 6 0 1 2 0 2 6 S e p

Claims

1. A plasmid comprising a nucleic acid sequence comprising nucleotides 1-4397 of SEQ ID NO: 7, nucleotides 1-4386 of SEQ ID NO: 18, nucleotides 1-4375 of SEQ ID NO: 8, or nucleotides 1-4364 of SEQ ID NO: 17.

2. The plasmid of claim 1, wherein said plasmid lacks AAV rep and cap genes.

3. The plasmid of claim 1 or claim 2 further comprising a selectable marker.

4. A host cell comprising the plasmid of any one of claims 1 to 3.

5. A packaging cell comprising the plasmid of any one of claims 1 to 4.

6. The packaging cell of claim 5 selected from the group consisting of HeLa cells, PerC.6 cells, 293 cells, low passage 293 cells, MRC-5 cells, WI-38 cells, Vero cells and FRhL-2 cells.

7. A method of producing a recombinant adeno-associated virus (rAAV) particle comprising transforming a cell with a plasmid, wherein the plasmid comprises a nucleotide sequence that is at least 90% identical to nucleotides 1 to 4397 of SEQ ID NO: 7, nucleotides 1 to 4386 of SEQ ID NO: 18, nucleotides 1 to 4375 of SEQ ID NO: 8, or nucleotides 1 to 4364 of SEQ ID NO: 17.

8. The method of claim 7, wherein the cell is selected from the group consisting of HeLa cells, 293 cells, PerC.6 cells, MRC-5 cells, WI-38 cells, Vero cells and FRhL-2 cells.

9. The method of claim 8 further comprising purifying a rAAV particle from the supernatant of the cell.

Images