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U7snRNAs
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Proof-of-concept for multiple AON delivery by a single U7snRNA vector to restore splicing defects in…
In vitro methylation of the U7 snRNP subunits Lsm11 and SmE by the PRMT5/MEP50/pICln methylosome.
U7 snRNA: A tool for gene therapy.
In vitro methylation of the U7 snRNP subunits Lsm11 and SmE by the PRMT5/MEP50/pICln methylosome.
Promising AAV.U7snRNAs vectors targeting DMPK improve DM1 hallmarks in patient-derived cell lines.
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AAV9 U7snRNA Gene Therapy to Treat Boys With DMD Exon 2 Duplications.
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