Pathological mechanism and antisense oligonucleotide-mediated rescue of a non-coding variant suppressing factor 9 RNA biogenesis leading to hemophilia B
Krooss S, Werwitzke S, Kopp J, Rovai A, Varnholt D, Wachs A, Goyenvalle A, Aarstma-Rus A, Ott M, Tiede A, Langemeier J, Bohne J
