FIELD: medicine.
SUBSTANCE: disclosed is a method for increasing vector titers based on adeno-associated virus 9 (AAV9) in transfected producer cells grown in culture. Disclosed are methods of treating X-linked adrenoleukodystrophy (X-ALD) in a subject in need thereof. Disclosed is a method of delivering representative of 1 superfamily D ATP-binding cassette (ABCD1) to a subject with X-ALD, comprising introducing into a subject a vector containing NM_000033.3.
EFFECT: proposed group of inventions provides the ABCD1 coding nucleic acid sequences for the X-ALD treatment.
11 cl, 4 tbl, 3 ex, 14 dwg
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