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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

Simon Krooss,Sonja Werwitzke,Johannes Kopp,Alice Rovai, Dirk Varnholt,Amelie S. Wachs,Aurelie Goyenvalle, Annemieke Aarstma-Rus,Michael Ott,Andreas Tiede,Joerg Langemeier,Jens Bohne

PLoS Genetics（2021）

引用 5|浏览22
关键词
RNA Phase Transitions
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