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公开(公告)号：US20230038479A1
公开(公告)日：2023-02-09
申请号：US17791169
申请日：2021-02-02
Applicant: University of Sheffield
Inventor： Mimoun Azzouz , Christopher Webster
Abstract: The present disclosure relates to transcription cassettes comprising nucleic acids encoding RuvBL1 and/or RuvBL2 and the use of said vectors in gene therapy for the treatment of neurodegenerative diseases that result from expression of polymorphic repeat expansions of the GGGGCC (SEQ ID NO: 5) hexanucleotide-repeat sequence in the first intron of the C9ORF72 gene; pharmaceutical compositions comprising said vectors and including uses and methods to treat neurodegenerative diseases.
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