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Pathological mechanism and antisense oligonucleotide-mediated rescue of a non-coding variant suppressing factor 9 RNA biogenesis leading to hemophilia B
by
Krooss, Simon
,
Werwitzke, Sonja
,
Kopp, Johannes
,
Rovai, Alice
,
Varnholt, Dirk
,
Wachs, Amelie S
,
Goyenvalle, Aurelie
,
Aarstma-Rus, Annemieke
,
Ott, Michael
,
Tiede, Andreas
,
Langemeier, Jörg
,
Bohne, Jens
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PLoS genetics
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Antisense Oligonucleotides
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