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filingDate 2019-05-24-04:00^^<http://www.w3.org/2001/XMLSchema#date>
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publicationDate 2019-10-03-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber WO-2019185067-A1
titleOfInvention APPLICATION OF LENTIVIRAL VECTOR EF1α PROMOTER FOR OPTIMISING ABCD1 GENE EXPRESSION TO TREAT ADRENOLEUKODYSTROPHY
abstract Provided is a lentiviral vector comprising an EF1α promoter, a normal ABCD1 gene and an NHP/TYF lentiviral vector system, said vector being used for treating adrenoleukodystrophy. The present invention uses transfection into autologous haematopoietic stem cells (HSCs), for ALD gene therapy after being returned, which may be performed in combination with direct intracerebral injection of the lentiviral vector carrying the ABCD1 gene according to the actual circumstances.
priorityDate 2018-03-29-04:00^^<http://www.w3.org/2001/XMLSchema#date>
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