Pathological mechanism and antisense oligonucleotide-mediated rescue of a non-coding variant suppressing factor 9 RNA biogenesis leading to hemophilia B
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
Published in PLoS genetics (01.04.2020)
Published in PLoS genetics (01.04.2020)
Get full text
Journal Article