基因编辑仓鼠Pathological mechanism and antisense oligonucleotide-mediated rescue of a non-coding variant suppressing factor 9 RNA biogenesis leading to hemophilia B.
期刊:PLoS genetics发表年份:2020作者:Simon Krooss; Sonja Werwitzke; Johannes Kopp; Alice Rovai; Dirk Varnholt; Amelie S Wachs; Aurelie Goyenvalle; Annemieke Aarstma-Rus; Michael Ott; Andreas Tiede; Jörg Langemeier; Jens Bohne
