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Authors
Dowling, James J. https://orcid.org/0000-0002-3984-4169
Pirovolakis, Terry
Devakandan, Keshini
Stosic, Ana
Pidsadny, Mia
Nigro, Elisa
Sahin, Mustafa https://orcid.org/0000-0001-7044-2953
Ebrahimi-Fakhari, Darius
Messahel, Souad
Varadarajan, Ganapathy
Greenberg, Benjamin M. https://orcid.org/0000-0002-2091-8201
Chen, Xin
Minassian, Berge A.
Cohn, Ronald https://orcid.org/0000-0002-6775-1496
Bonnemann, Carsten G. https://orcid.org/0000-0002-5930-2324
Gray, Steven J. https://orcid.org/0000-0002-6240-8621
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Article History
Received: 2 August 2023
Accepted: 20 May 2024
First Online: 28 June 2024
Competing interests
: S.J.G. and X.C. are inventors on a patent application for the AP4M1 vector design. Of note, T.P. is a parent of the study patient. Also, subsequent to the completion of this study, T.P. formed Elpida Therapeutics, and MELPIDA (AAV-AP4M1) represents one of the clinical programs in its developmental pipeline. S.J.G. is a nonpaid member of the Elpida board of directors, and S.M. is head of clinical operations. The other authors declare no competing interests.
