ABCD1 for treatment of neurodisorders

A method of increasing titers of an adeno-associated virus 9 (AAV9) vector comprising a nucleotide sequence encoding ATP binding cassette subfamily D member 1 (ABCD1) in transfected producer cells grown in culture, said method comprising the steps of:

• i) incubating a pooled combination of siRNAs consisting of SEQ ID NO:
• 4, SEQ ID NO:
• 5, SEQ ID NO:
• 6, and SEQ ID NO:
• 7, wherein each siRNA of the pooled combination of siRNAs is complementary to an mRNA encoding ABCD1 with the cells, wherein each of the siRNAs of the pooled combination of siRNAs are at an equal concentration, and wherein the pooled combination of siRNAs has a total concentration of 5 μM, and
• ii) transfecting the AAV9 vector comprising the nucleotide sequence encoding ABCD1 into the cells, wherein the amount of ABCD1 mRNA expressed from the AAV9 vector is decreased compared to an amount prior to the transfecting, thereby increasing AAV9-ABCD1 vector yield in cell lysate and/or media by up to 4 fold compared to yield in cell lysate and/or media from producer cells that were (1) transfected with the AAV9 vector comprising the nucleotide sequence encoding ABCD1 and (2) not incubated with the pooled combination of siRNAs consisting of SEQ ID NO:
• 4, SEQ ID NO:
• 5, SEQ ID NO:
• 6, and SEQ ID NO:
• 7,
• wherein the producer cells are selected from the group consisting of 293 cells, HeLa cells, and insect cells.