Secondary failure of lentiviral vector gene therapy in a cerebral adrenoleukodystrophy patient with an ABCD1 whole-gene deletion
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Published:2024-08
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ISSN:1525-0016
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Container-title:Molecular Therapy
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language:en
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Short-container-title:Molecular Therapy
Author:
Lund Troy C.ORCID,
Orchard Paul J.,
Nascene David R.,
King Carina J.,
Braun Jennifer,
Thakkar Stuti,
Durose Willa,
Shestopalov Ilya,
Thakar Himal,
Gupta Ashish O.